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.

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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.

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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.

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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.

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Regulus Therapeutics Announces Receipt of FDA Orphan Drug Designation for RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)
by BPG Blog

SAN DIEGO, June 21, 2022 /PRNewswire/ — Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the “Company” or “Regulus”), today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to RGLS8429 for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD).

“Patients living with ADPKD currently have limited treatment options and approximately half of patients develop end-stage renal disease by age 60 requiring dialysis or transplantation,” commented Jay Hagan, CEO of Regulus Therapeutics. “ADPKD is a disease of high unmet need affecting nearly 160,000 Americans. We look forward to advancing RGLS8429 through the clinic with the goal of improving future treatment options for patients in need.”

The FDA’s Office of Orphan Products Development grants orphan designation status 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 designation status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including financial incentives, to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.

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.

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Phanes Therapeutics’ PT217 receives Orphan Drug Designation for small cell lung cancer from the FDA
by BPG Blog

SAN DIEGO, June 21, 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 PT217 for the treatment of small cell lung cancer (SCLC).

PT217 is a first-in-class bispecific antibody targeting Delta-like ligand 3 (DLL3) and cluster of differentiation 47 (CD47) being developed for patients with SCLC and other neuroendocrine cancers.  SCLC is an aggressive pulmonary carcinoma hallmarked by high early mortality rates and significant morbidities throughout the disease’s progression. The 1-year survival of patients with SCLC is only 32.9%, with only 10.7% of patients surviving 3 years.

“PT217 has the potential to be a transformative treatment option for SCLC patients whose initial response to chemotherapy is short-lived and inevitably becomes resistant to chemotherapeutic agents” said Dr. Ming Wang, Founder and CEO of Phanes Therapeutics. “We have built a strong pipeline in immuno-oncology by leveraging our proprietary technology platforms and expect to file an IND for PT217 by the third quarter of this year. This orphan drug designation follows two recent IND clearances for our PT199, an anti-CD73 monoclonal antibody and PT886, an anti-Claudin 18.2/anti-CD47 bispecific antibody, programs which we are progressing into the clinic.”

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.

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LG Chem’s New Drug for Obesity Receives Additional Orphan Drug Designation by the US FDA
by BPG Blog

LG Chem announced on 16th that the FDA recently granted ODD to ‘LB54640’ for the treatment of ‘POMC (Proopiomelanocortin) deficiency.’

‘LB54640’ also received ODD back in September of 2020 by the FDA for ‘LEPR (leptin receptor) deficiency’.

The FDA operates the ODD system to encourage and support the development of rare and difficult-to-treat disease drugs with prevalence rates of less than 200,000 people. It is estimated that there are about 120,000 people suffering from rare genetic obesity in the US.

With this designation, LG Chem will receive benefits such as exclusive sales rights in the US market for seven years, subsidies and tax cuts for clinical trial expenses in the US, and preliminary consulting support related to development.

Click here to read the full article at Business Wire

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.

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ResVita Bio receives Rare Pediatric Disease Designation for RVB-001 as a Treatment for Netherton Syndrome
by BPG Blog

BERKELEY, Calif., June 9, 2022 /PRNewswire/ — ResVita Bio, a cellular therapies startup, announces that the FDA has granted Rare Pediatric Disease Designation to RVB-001 to treat Netherton Syndrome, a chronic and life-threatening genetic skin disease.

“The physical and emotional challenges of chronic skin diseases are devastating to patients, particularly children” said Amin Zargar, Chief Executive Officer of ResVita Bio. “Newborns with Netherton Syndrome face a severe lifelong disease with few treatment options that offer limited efficacy. We are motivated by the prospect to deliver meaningful improvement in the lives of these patients, and this is a significant step towards the development RVB-001 as a treatment for Netherton Syndrome.”

With the Rare Pediatric Disease Designation, if a new drug application for RVB-001 is approved, ResVita Bio may be eligible to receive a priority review voucher, which can be redeemed for priority review of any subsequent drug marketing application. The voucher can also be sold or transferred.

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 (4)

Vertex Kidney Disease Drug Scores FDA Breakthrough Therapy Designation
by BPG Blog

Vertex Pharmaceuticals announced on Wednesday that the U.S. Food and Drug Administration granted Breakthrough Therapy Designation to its candidate, inaxaplin (VX-147). Inaxaplin was granted the designation for the treatment of APOL1-mediated focal segment glomerulosclerosis (FSGS).

APOL1-mediated FSGS is a kidney disease in which scar tissue develops on parts of the kidney called glomeruli, which is associated with APOL1 genetic mutations. The APOL1 gene is associated with the innate immune system, and people who have two mutations in the gene are at an increased risk of developing kidney diseases such as FSGS.

This is due to increased toxicity of the APOL1 protein in the kidney, which is the result of inflammatory exposures. Currently, there are no approved treatments for the disease, although treatments such as high-dose steroids can delay the progression to kidney failure.

Click here to read the full article at BioSpace

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.

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Apellis and Sobi Announce First Patient Dosed in Phase 3 VALIANT Study of Pegcetacoplan for IC-MPGN and C3G, Rare Kidney Diseases with High Unmet Medical Need
by BPG Blog

WALTHAM, Mass. and STOCKHOLM, Sweden, June 07, 2022 (GLOBE NEWSWIRE) — Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Sobi® (STO:SOBI) today announced that the first patient has been dosed in the Phase 3 VALIANT study investigating pegcetacoplan, a targeted C3 therapy, in primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G), two rare and debilitating kidney diseases with similar underlying causes and no approved treatment.

“Individuals with IC-MPGN and C3G experience a high burden of disease due to the lack of approved treatments to slow or stop disease progression. These diseases often lead to kidney failure, requiring dialysis or a kidney transplant, imposing further substantial burdens,” said Kristen Hood, MSN, RN, executive director of research engagement at Nephcure Kidney International. “We are excited that Apellis and Sobi are advancing a Phase 3 study of pegcetacoplan in patients aged 12 and older, with primary IC-MPGN or C3G, including those with post-transplant recurrence.”

Uncontrolled activation of the complement cascade, a part of the body’s immune system, is believed to play a critical role in the progression of IC-MPGN and C3G where excessive accumulation of C3 breakdown products in the kidney causes inflammation and organ damage.1-4 It is estimated that 5,000 people in the United States and up to 8,000 in Europe are living with IC-MPGN or C3G,5 and approximately 50% ultimately suffer from kidney failure within five to 10 years of diagnosis.6

Click here to read the full article at FDANews

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.