BPG BLOG 42

Wugen Receives U.S. FDA Fast Track and Rare Pediatric Disease Designations for WU-CART-007 for the Treatment of R/R T-ALL/LBL
by BPG Blog

ST. LOUIS & SAN DIEGO–(BUSINESS WIRE)–Wugen, Inc., a clinical-stage biotechnology company developing a pipeline of allogeneic cell therapies to treat a broad range of hematological and solid tumor malignancies, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation and Rare Pediatric Disease Designation (RPDD) for WU-CART-007, the company’s off-the-shelf CAR-T cell therapy. WU-CART-007 is currently being explored for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL).

“We are very pleased to have received both fast track and rare pediatric disease designations, which re-affirm the great unmet need for new treatment options for people with R/R T-ALL/LBL,” said Dan Kemp, Ph.D., President and Chief Executive Officer of Wugen. “Earlier this year, we dosed the first patient in our ongoing Phase 1/2 trial of WU-CART-007 for R/R T-ALL/LBL and are currently in the dose escalation phase of the study. We look forward to working closely with the FDA as we continue to advance WU-CART-007 through clinical development.”

The FDA’s Fast Track is a process designed by the FDA to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. According to the FDA, the purpose is to get important new drugs to the patient earlier. Features of Fast Track Designation include opportunities for more frequent interactions with the FDA review team and, if supported by clinical data, eligibility for Priority Review.

Click here to read the full article at Cision PR Newswire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG 2

AVROBIO Receives Orphan Drug Designation from the U.S. Food and Drug Administration for AVR-RD-05, a Gene Therapy for Mucopolysaccharidosis Type II (MPSII) or Hunter Syndrome
by BPG Blog

CAMBRIDGE, Mass.–(BUSINESS WIRE)–AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for AVR-RD-05, its gene therapy for the treatment of mucopolysaccharidosis type II (MPSII), or Hunter syndrome, a rare and seriously debilitating lysosomal disorder that primarily affects young boys.

Orphan drug designation is granted by FDA to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain incentives, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers.

Hunter syndrome is caused by a deficiency in the lysosomal enzyme iduronate-2-sulfatase (IDS), which is essential for breaking down large sugar molecules. The gene therapy uses a patient’s own hematopoietic stem cells (HSCs) that are transduced ex vivo with a lentiviral vector encoding the human IDS enzyme. The company’s planned collaborator-sponsored Phase 1/2 clinical trial for Hunter syndrome is expected to commence in 2023 under the company’s collaboration with the University of Manchester, UK. The program was developed by Brian Bigger, Ph.D., professor of cell and gene therapy at the University of Manchester, who has previously published preclinical data demonstrating that HSC gene therapy deploying an optimized, proprietary tag has the potential to correct peripheral disease and normalize brain pathology.

Click here to read the full article at Cision PR Newswire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG (2)

Anthos Therapeutics Announces that Abelacimab has Received FDA Fast Track Designation for the Treatment of Thrombosis Associated with Cancer
by BPG Blog

CAMBRIDGE, Mass., July 11, 2022 /PRNewswire/ — Anthos Therapeutics, a clinical-stage biotechnology company developing innovative therapies for cardiovascular and metabolic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its investigational Factor XI inhibitor, abelacimab, for the treatment of thrombosis associated with cancer. The company will also be announcing this important milestone today at a session of the ongoing 2022 Congress of the International Society on Thrombosis and Haemostasis (ISTH) Congress in London, UK.

The Fast Track Designation process is designed to facilitate the development and expedite the review of treatments for serious medical conditions, thereby, addressing unmet medical needs. Drugs that are included in this program may be eligible for more frequent interactions with the FDA to discuss the development path, and if the program criteria are met, eligibility for a potential Rolling Review, Accelerated Approval, and Priority Review.

Venous Thromboembolism (VTE), including both deep vein thrombosis and pulmonary embolism, is the second most prevalent cause of death in patients with cancer, second only to the disease itself.1 However, treatment of Cancer Associated Thrombosis (CAT) can be challenging because the currently available anticoagulants used to treat VTE can have an increased risk of bleeding.2,3

Click here to read the full article at Cision PR Newswire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG highlight

Rare Disease Highlight: Alternating Hemiplegia of Childhood
by Rare Disease Highlight

Alternating Hemiplegia of Childhood (AHC) is a rare and severe neurodevelopmental disorder that fully manifests during early childhood, typically within the first year of life. AHC is characterized by repeated attacks of hemiplegia, paralysis of one side of the body, or episodes of quadriplegia, simultaneous paralysis of both sides of the body (Heinzen et al., 2014; NORD, 2016; Panagiotakaki et al., 2010; Rosewich et al., 2017; Sweney et al., 2009). The duration of the episodes of paralysis vary, but can last for minutes up to weeks at a time in some cases (NORD, 2016). AHC occurs in approximately 1 in every 1,000,000 births (NORD, 2016).

The symptoms of AHC are diverse and can vary greatly between patients (NORD, 2016). The key hallmark of AHC is the appearance of recurring episodes of paralysis, known as plegic episodes (either hemiplegia or quadriplegia). Other symptoms include tonic attacks (lack of muscle tone), dystonia (painful involuntary muscle contractions), ataxia (lack of coordination), eye problems including nystagmus (uncontrollable eye movements) and strabismus (misalignment of the eyes), developmental delays, neurologic abnormalities, difficulty breathing (dyspnea), and seizures (AHCF, 2022; Panagiotakaki et al., 2010; Sweney et al., 2009). Patients can also develop epilepsy (a brain disorder that causes recurrent and unprovoked seizures) as they get older (NORD, 2016). Symptoms are episodic, range in severity and duration, from minutes to days, and are typically relieved with sleep (AHCF, 2022; Sweney et al., 2009). AHC attacks are triggered by environmental stress, water exposures, specific physical activities, lighting changes, and certain foods (AHCF, 2022; Sweney et al., 2009). During the plegic attacks, patients remain conscious, so the attacks are accompanied by painful vocalization and respiratory compromise (Panagiotakaki et al., 2010; Save et al., 2013; Silver et al., 1993). The prevalence of plegic attacks decreases moderately with age, however these episodic events persist into adulthood (Panagiotakaki et al., 2010).

The cause of AHC is the topic of much research and investigation. In most patients, approximately 76%, AHC is associated with a new sporadic mutation in the ATP1A3 gene (AHCF, 2022; Heinzen et al., 2014; Rosewich et al., 2017). This gene produces the Sodium-Potassium-Transporting ATPase Subunit Alpha 3 protein, required for the normal functioning of neurons (brain nerve cells). Loss of ATPase activity is the root cause of AHC. However, some AHC patients do not present mutations in the ATP1A3 gene, thus scientists believe mutations in other genes could also be associated with AHC onset. Even though AHC appears due to genetic mutations, it is not commonly transmitted from parents to their children. However, in rare cases, AHC can run in families (NORD, 2016).

Patients with AHC experience a reduced quality of life. Patients suffer complications including epilepsy, cognitive impairment, persistent movement disorder, developmental delays, learning disabilities, behavioral issues, sleep disorders, and motor deterioration, all of which appear during childhood and remain into adulthood (Duke Health, 2021; Mikati et al., 2000; Sweney et al., 2009). While life expectancy is not generally affected, children with AHC are at higher risk of life-threatening complications such as breathing difficulties (AHCF, 2022).There are currently no FDA-approved therapies for the treatment of AHC. However, symptoms of AHC can be managed by avoiding triggers and using sleep as a management tactic. AHC attacks and epileptic episodes may also be treated with pharmacologic intervention. The only drug proven effective in reducing the frequency and severity of AHC attacks is the calcium channel entry blocker Flunarizine (approved in Canada). However, it is not effective in all cases and is not readily available in the US (AHCF, 2022; Duke Health, 2021; Kansagra et al., 2013; Neville et al., 2007; NORD, 2016). Therefore, there exists an urgent unmet medical need for patients and caregivers burdened by the profound morbidities that accompany AHC. The development of a therapy administered soon after diagnosis that prevents or reduces the frequency of plegic and dystonic attacks may improve long-term outcomes of patients over the course of their lifetime.

References

AHCF. (2022). Alternating Hemiplegia of Childhood Foundation – What is AHC? Retrieved July 12, 2022 from http://ahckids.org/education/whatisahc/

Duke Health. (2021). Alternating Hemiplegia of Childhood. Retrieved July 12, 2022 from https://www.dukehealth.org/treatments/pediatric-neurology/alternating-hemiplegia-of-childhood

Heinzen, E. L., Arzimanoglou, A., Brashear, A., Clapcote, S. J., Gurrieri, F., Goldstein, D. B., Jóhannesson, S. H., Mikati, M. A., Neville, B., Nicole, S., Ozelius, L. J., Poulsen, H., Schyns, T., Sweadner, K. J., van den Maagdenberg, A., & Vilsen, B. (2014). Distinct neurological disorders with ATP1A3 mutations. Lancet Neurol, 13(5), 503-514. doi:10.1016/s1474-4422(14)70011-0

Kansagra, S., Mikati, M. A., & Vigevano, F. (2013). Alternating hemiplegia of childhood. Handb Clin Neurol, 112, 821-826. doi:10.1016/b978-0-444-52910-7.00001-5

Mikati, M. A., Kramer, U., Zupanc, M. L., & Shanahan, R. J. (2000). Alternating hemiplegia of childhood: clinical manifestations and long-term outcome. Pediatr Neurol, 23(2), 134-141. doi:10.1016/s0887-8994(00)00157-0

Neville, B. G., & Ninan, M. (2007). The treatment and management of alternating hemiplegia of childhood. Dev Med Child Neurol, 49(10), 777-780. doi:10.1111/j.1469-8749.2007.00777.x

NORD. (2016). National Organization for Rare Diseases – Alternating Hemiplegia of Childhood. Retrieved July 12, 2022 from https://rarediseases.org/rare-diseases/alternating-hemiplegia-of-childhood/

Panagiotakaki, E., Gobbi, G., Neville, B., Ebinger, F., Campistol, J., Nevsímalová, S., Laan, L., Casaer, P., Spiel, G., Giannotta, M., Fons, C., Ninan, M., Sange, G., Schyns, T., Vavassori, R., Poncelin, D., & Arzimanoglou, A. (2010). Evidence of a non-progressive course of alternating hemiplegia of childhood: study of a large cohort of children and adults. Brain, 133(Pt 12), 3598-3610. doi:10.1093/brain/awq295

Rosewich, H., Sweney, M. T., DeBrosse, S., Ess, K., Ozelius, L., Andermann, E., Andermann, F., Andrasco, G., Belgrade, A., Brashear, A., Ciccodicola, S., Egan, L., George, A. L., Jr., Lewelt, A., Magelby, J., Merida, M., Newcomb, T., Platt, V., Poncelin, D., Reyna, S., Sasaki, M., Sotero de Menezes, M., Sweadner, K., Viollet, L., Zupanc, M., Silver, K., & Swoboda, K. (2017). Research conference summary from the 2014 International Task Force on ATP1A3-Related Disorders. Neurol Genet, 3(2), e139. doi:10.1212/nxg.0000000000000139

Save, J., Poncelin, D., & Auvin, S. (2013). Caregiver’s burden and psychosocial issues in alternating hemiplegia of childhood. European Journal of Paediatric Neurology, 17(5), 515-521. doi:https://doi.org/10.1016/j.ejpn.2013.04.002

Silver, K., & Andermann, F. (1993). Alternating hemiplegia of childhood: a study of 10 patients and results of flunarizine treatment. Neurology, 43(1), 36-41. doi:10.1212/wnl.43.1_part_1.36

Sweney, M. T., Silver, K., Gerard-Blanluet, M., Pedespan, J.-M., Renault, F., Arzimanoglou, A., Schlesinger-Massart, M., Lewelt, A. J., Reyna, S. P., & Swoboda, K. J. (2009). Alternating Hemiplegia of Childhood: Early Characteristics and Evolution of a Neurodevelopmental Syndrome. Pediatrics, 123(3), e534-e541. doi:10.1542/peds.2008-2027

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG 3

Phanes Therapeutics’ PT886 granted Orphan Drug Designation for the treatment of pancreatic cancer by the FDA
by BPG Blog

SAN DIEGO, June 30, 2022 /PRNewswire/ — Phanes Therapeutics, Inc. (Phanes), an emerging leader in innovative discovery research and clinical development in oncology, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PT886 for the treatment of pancreatic cancer. Earlier this month, the FDA granted orphan drug designation to the company’s PT217 for the treatment of small cell lung cancer.

PT886 is a first-in-class bispecific antibody targeting claudin 18.2 (CLDN18.2) and cluster of differentiation 47 (CD47) being developed for patients with pancreatic cancer as well as gastric and gastroesophageal cancers. Pancreatic cancer is an aggressive form of cancer characterized by high mortality rates and significant morbidities. The 5-year survival rate of patients with pancreatic cancer is only 10.8% and projections for 2022 estimate that approximately 50,000 Americans will die of pancreatic cancer this year and by 2030 is projected to exceed breast, prostate, and colorectal malignancies as the leading cause of cancer-related deaths in the US.

“PT886 has the potential to be a transformative treatment option for pancreatic cancer patients whose current standard of care is severely limited.” said Dr. Ming Wang, Founder and CEO of Phanes Therapeutics. “This orphan drug designation comes in the same month with our recent IND clearance for PT886, which we are rapidly progressing into the clinic. These important milestones the company has achieved in Q2 this year follow the March IND clearance for PT199, an anti-CD73 monoclonal antibody for the treatment of multiple solid tumors.”

Click here to read the full article at Cision PR Newswire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG 28

Aura Biosciences Receives FDA Fast Track Designation for Belzupacap Sarotalocan (AU-011) for the Treatment of Non-Muscle Invasive Bladder Cancer
by BPG Blog

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for belzupacap sarotalocan (AU-011), Aura’s first VDC product candidate, for the treatment of Non-Muscle Invasive Bladder Cancer (NMIBC).

“We are pleased that belzupacap sarotalocan has been granted Fast Track designation. We believe that, given that NMIBC presents such a high unmet medical need, the opportunity for more frequent interactions with Division of Oncology at FDA and the potential for Priority Review will be valuable as we advance further into clinical development in patients with NMIBC,” said Dr. Mark De Rosch, Chief Operating Officer and Head of Regulatory Affairs of Aura.

“NMIBC has no approved targeted therapies, and patients experience high levels of recurrence and progression, ultimately leading to cystectomy or the removal of the entire bladder,” said Dr. Cadmus Rich, Chief Medical Officer and Head of R&D of Aura. “This milestone supports our goal to advance the development of belzupacap sarotalocan for patients with NMIBC in need of better and earlier targeted treatment options with the potential to help preserve the bladder.”

Click here to read the full article at BusinessWire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG

ALX Oncology Receives U.S. FDA Orphan Drug Designation for Evorpacept for the Treatment of Patients with Acute Myeloid Leukemia
by BPG Blog

SOUTH SAN FRANCISCO, Calif., June 29, 2022 (GLOBE NEWSWIRE) — ALX Oncology Holdings Inc., (“ALX Oncology”) (Nasdaq: ALXO) a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, today announced that the U.S. Food and Drug Administration (“FDA”) granted orphan drug designation (“ODD”) to evorpacept, a next-generation CD47 blocker, for the treatment of patients with acute myeloid leukemia (“AML”).

“Receiving orphan drug designation in AML, and previously in gastric cancer, from the FDA is an important regulatory milestone and reflects the FDA’s recognition of evorpacept’s potential to improve clinical outcomes in patients with these advanced cancers,” said Sophia Randolph, M.D., Ph.D., Chief Medical Officer, ALX Oncology. “In our ongoing Phase 1/2 ASPEN-05 study (NCT04755244), we are excited to evaluate the combination of evorpacept with venetoclax and azacitidine in patients with previously untreated AML who are not candidates for intensive induction therapy or with relapsed/refractory AML.”

The FDA’s Office of Orphan Products Development grants ODD status to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. ODD provides benefits to drug developers designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include assistance in the drug development process, tax credits for qualified clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.

Click here to read the full article at BusinessWire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG (10)

Apnimed Granted FDA Fast Track Designation for AD109, a Novel First-in-Class Oral Pharmacologic Combination for the Treatment of Obstructive Sleep Apnea (OSA)
by BPG Blog

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Apnimed, a clinical-stage pharmaceutical company focused on developing oral pharmacologic treatments to address obstructive sleep apnea (OSA) and related disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its oral pharmacologic AD109 for the treatment of OSA.

“Fast Track designation is a significant milestone in the development of AD109 and provides an accelerated regulatory pathway that recognizes the urgent need for new pharmacologic treatments for OSA that are easier for people to tolerate,” said Larry Miller, M.D., Chief Executive Officer of Apnimed. “Currently the vast majority of more than 35 million Americans who have OSA remain untreated despite the potential for serious health risks associated with the condition, including cardiovascular disease and diabetes. We will continue to work closely with the FDA to support the development and review of AD109 beginning with the trial design for our Phase 3 program, which we anticipate initiating at the end of 2022.”

FDA’s Fast Track designation is intended to facilitate the development and expedite the review of new drugs to treat serious conditions and that fill an unmet medical need. The benefits of Fast Track designation include opportunities for frequent meetings with the FDA to discuss development plans, trial design, and data needed to support drug approval, as well as the ability to submit a New Drug Application (NDA) on a rolling basis, and eligibility for priority review, if relevant criteria are met.

Click here to read the full article at BusinessWire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG (9)

BioMarin Receives Positive CHMP Opinion in Europe for Valoctocogene Roxaparvovec Gene Therapy to Treat Adults with Severe Hemophilia A
by BPG Blog

SAN RAFAEL, Calif., June 24, 2022 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in Q3 2022.

The one-time infusion is planned to be marketed under the brand name ROCTAVIAN™ (valoctocogene roxaparvovec), for the treatment of severe hemophilia A (congenital factor VIII deficiency) in adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).  Roctavian is the first gene therapy to be recommended for approval in Europe for hemophilia A.  

It is estimated that more than 20,000 adults across Europe, Middle East, and Africa are affected by severe hemophilia A. BioMarin anticipates additional patient access through named patient sales based on an EMA approval in countries in the Middle East and Africa and expects additional market registrations to be facilitated by an anticipated EMA license.

Click here to read the full article at Cision PR Newswire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

BPG BLOG (8)

Adverum Biotechnologies Granted Priority Medicines (PRIME) Designation by European Medicines Agency for ADVM-022 in Wet AMD
by BPG Blog

REDWOOD CITY, Calif., June 24, 2022 (GLOBE NEWSWIRE) — Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced that the European Medicines Agency (EMA) has granted ADVM-022 Priority Medicines (PRIME) designation for the treatment of wet age-related macular degeneration (wet AMD).  Adverum’s lead gene therapy candidate, ADVM-022 is a one-time, intravitreal (IVT) injection for the treatment of patients with wet AMD.

PRIME is a program launched by the EMA to enhance support for research on and development of medicines that target a significant unmet medical need. This regulatory program offers developers of promising medicines enhanced interaction and early dialogue and is designed to optimize development plans and speed evaluation ensuring these medicines reach patients as early as possible. According to the EMA, developers of medicines that are eligible for PRIME can expect additional opportunities for scientific advice and be eligible for accelerated assessment at the time of application for a marketing authorization. Currently, only three ophthalmology therapies to date have been granted PRIME designation out of 14 submissions.

“We are extremely pleased with EMA’s decision to grant ADVM-022 PRIME designation. Following the encouraging results seen in the OPTIC trial in wet AMD, our PRIME designation recognizes the broad potential of ADVM-022 and is based in part on our trial subjects now having three-year sustained aflibercept expression with stable disease and maintained long-term central subfield thickness and visual acuity after a single IVT injection,” said Laurent Fischer, M.D., president and chief executive officer at Adverum Biotechnologies. “The current standard of care in wet AMD, a highly prevalent disease, requires frequent anti-vascular endothelial growth factor (anti-VEGF) injections in the eye and is a lifelong burden for many patients and their caregivers. We believe ADVM-022 has the potential to provide a durable, safe and cost-effective in-office treatment option that addresses the needs of these patients, and their families, as well as retina specialists and health systems worldwide. We look forward to taking advantage of the benefits provided to us through PRIME as we continue the development of ADVM-022 in areas of high unmet need.”

Click here to read the full article at GlobeNewswire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.