03102021

Oblato Announces Fast Track Designation of OKN-007 for Diffuse Intrinsic Pontine Glioma from the FDA

Oblato, Inc. (the Company), a wholly owned U.S. subsidiary of the Korean biotech company GtreeBNT Co., Ltd., announces that the FDA granted Fast Track Designation of OKN-007, the proprietary drug for Diffuse Intrinsic Pontine Glioma (DIPG).

The FDA Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

With the Fast Track Designation of OKN-007 for the treatment of DIPG, Oblato is eligible for Accelerated Approval and Priority Review and for Rolling Review that allows the Company to submit completed sections of its New Drug Application (NDA) for review by FDA before the entire application can be reviewed. The Company expects that the period of the regulatory approval process will be reduced.

DIPG is a rare pediatric brainstem cancer with limited treatment options. After a DIPG diagnosis, ninety percent of the patients die within 24 months, and the overall 5-year survival is less than one percent.

In addition to the Fast Track Designation, Oblato has already received Rare Pediatric Disease designation for DIPG. The Company is eligible for a rare pediatric disease priority review voucher from the FDA that can be redeemed for a priority review of a marketing application of a different product and is transferrable, when the Company receives NDA approval.

“We anticipate that the Fast Track Designation for DIPG will shorten the NDA review process. The Company will make every effort to develop this treatment for this rare pediatric disease,” stated an official from the Company.

To Read the Complete Article at PR Newswire, Click Here

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, operating like a not-for-profit, 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 high unmet medical need. 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.

Stock image by @vitanovski from Depositphotos

03092021

FDA Granted Orphan Drug Designation to Novel ImPACT Agent in Upper Tract Urothelial Cancer

The FDA has granted an Orphan Drug Designation to Padeliporfin Immune Photo Activated Cancer Therapy (ImPACT) for the treatment of adult patients with upper tract urothelial cancer, according to a press release from Steba Biotech.

“We are delighted to receive Orphan Drug Designation for Padeliporfin ImPACT in upper tract urothelial cancer, further validating Steba’s technology and the benefits our product could bring to people suffering from upper tract urothelial cancer. It has been less than a year since we embarked on our new strategy, refocusing Padeliporfin ImPACT on diseases for patients with limited treatment options,” said Barack Palatchi, chief executive officer, Steba Biotech, in a statement.

Following an Investigation New Drug Application, which was accepted by the FDA, a phase 3 clinical trial (ENLIGHTED; NCT04620239) has been initiated to further study the use of Padeliporfin ImPACT. Enrollment is expected to begin later this month.

ENLIGHTED is a single-arm, non-randomized, multicenter study with a target enrollment of 100 patients with low-grade upper tract urothelial cancer. The primary end point of the study is the number of patients with the absence of upper tract urothelial cancer tumors in the entire ipsilateral renal pelvis and ureter. The secondary end points of the study include the duration of response at the entire ipsilateral kidney, at the entire ipsilateral kidney, and at the treatment area of the ipsilateral kidney; overall renal function; the number of patients with kidney organ loss or preservation; and the number of patients with ureteral obstruction.

Padeliporfin ImPACT is an endoluminal light-activated treatment that involves the placement of optical light fiber with a ureteroscope and intravenous administration of Padeliporfin at 4 mg/kg over 10 minutes and the illumination of each area for 10 minutes in the study. The treatment triggers the constriction of the blood supply in the affected area upon activation and leads to targeted tumor necrosis and antitumor immunity.

To Read the Complete Article at Targeted Oncology, Click Here

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, operating like a not-for-profit, 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 high unmet medical need. 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.

Stock image by @Giovanni_Cancemi from Depositphotos

03082021

MedAlliance Receives Fourth FDA Breakthrough Device Designation for Sirolimus Drug-Eluting Balloon in Treatment of De Novo Coronary Lesions

MedAlliance, the first drug-eluting balloon (DEB) company in the world to receive US Food and Drug Administration (FDA) Breakthrough Device Designation Status for a sirolimus DEB, has now been awarded breakthrough status for SELUTION SLR™, its sustained limus release DEB catheter, in the treatment of atherosclerotic lesions in native coronary arteries. This is the fourth breakthrough designation awarded to MedAlliance for its sirolimus DEB, following coronary in-stent restenosis, peripheral below-the-knee and AV-Fistula indications.

According to the FDA, the SELUTION SLR 014 DEB Breakthrough Designation is for improving luminal diameter in patients with atherosclerotic lesions in native coronaries.

“MedAlliance is honoured to have our sirolimus DEB selected for the FDA’s Breakthrough Device Program for a fourth time. This may provide US patients faster access to our novel 90 day sustained sirolimus release technology, with the potential to provide safer and more effective treatment,” said Jeffrey B. Jump, Chairman and CEO of MedAlliance. “This Designation, combined with the previous coronary ISR Breakthrough Designation, will give US cardiologists exciting new tools to fight coronary disease.”

The FDA Breakthrough Device Program is intended to help patients receive more timely access to breakthrough technologies that have the potential to provide more effective treatment or diagnosis for life-threatening or irreversibly debilitating diseases or conditions. Under the program, the FDA will provide MedAlliance with priority review and interactive communication regarding device development and clinical trial protocols, through to commercialization decisions.

To Read the Complete Article at BioSpace, Click Here

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, operating like a not-for-profit, 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 high unmet medical need. 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.

Stock image by @CLIPAREA from Depositphotos

03052021

CARsgen Therapeutics Receives Orphan Medicinal Product Designation from the European Medicines Agency for CT041 CLDN18.2 CAR T Cells for the Treatment of Gastric Cancers

CARsgen Therapeutics Holdings Limited, a clinical-stage biopharmaceutical company, today announced that the European Commission (EC) has granted orphan designation for CT041, CARsgen’s first-in-class Claudin 18.2 (CLDN18.2) targeted CAR-T product candidate for the treatment of gastric cancer. CT041 consists of the patient’s own T cells, genetically modified to express a humanized anti-CLDN18.2 chimeric antigen receptor (CAR) to treat patients with CLDN18.2-positive tumors.

“The orphan medicinal product designation of CT041 by the EC is another important recognition of CARsgen’s commitment to the development of CAR T-cell treatment for patients with advanced gastric cancer,” said Dr. Zonghai Li, founder, CEO and CSO of CARsgen. “According to the World Health Organization, over one million new cases of gastric adenocarcinoma are expected each year, and it is the seventh most prevalent cancer type worldwide [1]. Despite the development of novel therapies, gastric cancer is still a disease with one of the highest unmet medical needs. We reaffirm our long-standing commitment to cancer patients worldwide by expanding upon our CT041 clinical trial data to advance novel, safe and effective immunotherapies.”

The EC grants orphan drug designation to investigational treatments for rare conditions, those affecting fewer than five in 10,000 people in the European Union. Treatments that meet the European Medicines Agency’s orphan designation criteria qualify for incentives to encourage advancement of drug development.

CT041 is the first CLDN18.2-targeted CAR T-cell treatment that has received Investigational New Drug (IND) clearance by the US Food and Drug Administration (FDA) and the National Medical Products Administration (NMPA) in China. Three open-label, multicenter, Phase 1b clinical trials (NCT04404595, NCT04581473, and NCT03874897) to evaluate the safety and efficacy of autologous CT041 cell treatment in patients with advanced gastric, gastroesophageal, or pancreatic adenocarcinoma are currently underway.

To Read the Complete Article at PR Newswire, Click Here

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, operating like a not-for-profit, 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 high unmet medical need. 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.

Stock image by @SergeyNivens from Depositphotos

03042021

Anuncia Inc. Receives FDA “Breakthrough Device Designation” for ReFlow™ System Mini

Anuncia Inc., an emerging leader in Cerebral Spinal Fluid (CSF) management, received U.S. Food and Drug Administration (FDA) Breakthrough Device Designation for its ReFlow™ System Mini intended for the treatment of CSF disorders requiring shunting such as hydrocephalus, a debilitating and life-threatening condition affecting more than 1 million U.S. patients. Elsa Abruzzo, President of Anuncia Inc., stated, “Our team is very pleased to achieve this significant milestone making the ReFlow System Mini eligible for prioritized FDA regulatory review and Centers for Medicare and Medicaid (CMS) Medicare Coverage of Innovation Technology (MCIT) reimbursement review.”

As many as half of all intracranial shunts fail in the first two years, mainly due to blockages that impair CSF flow requiring emergency revision surgery, each costing upwards of $40,000. The breakthrough indication would allow in-clinic or at-home prophylactic flushing noninvasively by trained patients, caregivers, and clinicians to potentially prevent blockages. “The ReFlow System Mini is optimized to serve a broad hydrocephalus population—infants with non-communicating hydrocephalus, young women with pseudotumor cerebri, and older patients with communicating, idiopathic normal pressure hydrocephalus (iNPH). iNPH is known as the treatable dementia and is often misdiagnosed as Alzheimer’s or Parkinson’s,” commented Mark Luciano, MD, Ph.D., Director of the Cerebral Fluid Center and Professor of Neurosurgery at John Hopkins University in Baltimore.

In a preliminary study of patients at risk for shunt occlusion by two US pediatric centers of excellence, all intracranial shunts remained patent beyond one year using prophylactic flushing with the current ReFlow System. More than half of the patients are nearing their two-year, occlusion-free shunt anniversary. Ramin Eskandari, MD MS, Chief of Pediatric Neurosurgery at the Medical University of South Carolina, remarked, “The ReFlow System Mini is unique in that it could be used with any newly implanted or revised shunt system in any size patient. Coupled with at-home, noninvasive prophylatic flushing, the ReFlow System Mini may decrease emergent hospitalizations, reduce patient and healthcare system costs, and improve the everyday quality of life and overall clinical outcomes for these vulnerable patients living with hydrocephalus.”

To Read the Complete Article at PR Newswire, Click Here

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, operating like a not-for-profit, 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 high unmet medical need. 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.

Stock image by @poznyakov from Depositphotos

03032021

FDA Approves First Treatment for Rare Genetic Metabolic Pediatric Disorder

The disorder known as molybdenum cofactor deficiency (MoCD) Type A presents shortly after birth, often with severe encephalopathy and intractable seizures.

The FDA approved the first therapy for an ultra-rare genetic metabolic disorder that affects infants, who don’t normally live past the age of 4.

The disorder is known as molybdenum cofactor deficiency (MoCD) Type A, which presents shortly after birth, often with severe encephalopathy and intractable seizures.

The approval for the injection, fosdenopterin (Nulibry), was granted to BridgeBio Pharma and its affiliate, Origin Biosciences.

A progressive disease, MoCD Type A affects fewer than 150 patients across the globe. It is characterized by an inability to produce cyclic pyranopterin monophosphate (cPMP). The injection replaces missing cPMP in treated patients.

“Today’s action marks the first FDA approval for a therapy to treat this devastating disease,” Hylton V. Joffe, MD, MMSc, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research, said in a statement. “The FDA remains committed to facilitating the development and approval of safe and effective therapies for patients affected by rare diseases—an area of critical need.”

To Read the Complete Article at AJMC, Click Here

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, operating like a not-for-profit, 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 high unmet medical need. 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.

Stock image by @everythingposs from Depositphotos

03022021

AVROBIO Receives Orphan Drug Designation from the European Commission for AVR‑RD‑04, an Investigational Gene Therapy for Cystinosis

AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the European Commission (EC) has granted orphan drug designation for AVR-RD-04, the company’s investigational gene therapy for the treatment of cystinosis. AVR-RD-04 consists of the patient’s own hematopoietic stem cells, genetically modified to express cystinosin, the protein that is deficient in patients with cystinosis. AVR-RD-04 is currently being evaluated in a Phase 1/2 clinical trial (NCT03897361) sponsored by AVROBIO’s academic collaborator at the University of California, San Diego.1

“People with cystinosis must often adhere to an extremely challenging treatment regimen to manage symptoms of their disease. Despite this chronic treatment, many face a significantly shortened life expectancy and require major interventions, such as a kidney transplant,” said Geoff MacKay, president and CEO of AVROBIO. “This investigational gene therapy, delivered in a single dose, is designed to enable patients to endogenously produce the protein their cells need to prevent the toxic build-up of cystine in tissues throughout the body. We’re pleased to receive orphan drug designation in recognition of the potential of this approach to improve on the standard of care for people living with this relentless lysosomal disorder.”

The EC grants orphan drug designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and 10 years of market exclusivity following regulatory approval.

To Read the Complete Article at Business Wire, Click Here

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, operating like a not-for-profit, 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 high unmet medical need. 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.

Stock image by @Giovanni_Cancemi from Depositphotos

02252021

Immunocore’s TCR Bispecific Earns Breakthrough Designation for Ocular Cancer

Hot on the heels of its $297 million IPO earlier this month, British T cell receptor company Immunocore announced a U.S. Food and Drug Administration (FDA) Breakthrough Therapy Designation for its lead asset tebentafusp, currently in Phase III testing for a rare ocular melanoma. The company is planning regulatory filings in the U.S. later this year and in Europe thereafter.

Last year, Immunocore announced positive interim data for its Phase III trial in metastatic uveal melanoma (mUM) for tebentafusp, which is a soluble gp100-targeting T-cell receptor fused to an anti-CD3 single-chain variable fragment. The bispecific improved overall survival compared with the investigator’s choice of therapy, in most cases Merck & Co’s Keytruda. Keytruda is approved for cutaneous forms of metastatic melanoma and is regularly used to treat mUM, but no drug has been approved specifically for the rare subtype, and it has only shown efficacy for a limited number of responders in small clinical trials.

The new FDA designation means tebentafusp will remain in FDA’s Fast Track program and represents a commitment to rolling and priority drug reviews for HLA-A*02:01-positive adults with unresectable uveal melanoma or mUM. Immunocore intends to file a Biologics License Application in Q3, upon completion of its Phase III study, followed by a Marketing Authorization Application submission to the European Medicines Agency. The drug also has the Promising Innovative Medicine designation under the UK Early Access to Medicines Scheme.

“There is an urgent need for an approved treatment for this rare and aggressive form of melanoma,” said Bahija Jallal, Immunocore’s CEO. “We look forward to continuing to work with regulators to bring tebentafusp to patients as quickly as possible.” The company estimates no drug has been approved for mUM in 40 years.

To Read the Complete Article at BioSpace, Click Here

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, operating like a not-for-profit, 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 high unmet medical need. 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.

Stock image by @VadimVasenin from Depositphotos

02232021

Celsion Corporation Receives FDA Fast Track Designation for GEN-1 in Advanced Ovarian Cancer

Celsion Corporation (NASDAQ: CLSN), a clinical stage development company focused on DNA based immunotherapy and next generation vaccines, today announced that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for GEN-1, its DNA-mediated interleukin-12 (IL-12) immunotherapy currently in Phase II development for the treatment of advanced ovarian cancer. GEN-1 was designed using TheraPlas, Celsion’s proprietary, synthetic, non-viral nanoparticle delivery system platform.

Fast Track designation is intended to facilitate the development and expedite the regulatory review of drugs to treat serious conditions and fill an unmet medical need. According to the FDA, a Fast Track Drug must show some advantage over available therapy, including:

  • Showing superior effectiveness, effect on serious outcomes or improved effect on serious outcomes
  • Avoiding serious side effects of an available therapy
  • Decreasing a clinical significant toxicity of an available therapy that is common and causes discontinuation of treatment

“Fast Track designation is an important step in developing GEN-1 for advanced ovarian cancer. Presuming the encouraging data we are generating in early clinical studies continues, this designation supports an expedited path to market,” said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. “Fast Track allows for more frequent communication with the FDA to discuss development plans and clinical trial design. In addition, should criteria be met, Fast Track-designated drugs are eligible for rolling review, a process whereby the drug’s sponsor can separately submit sections of its New Drug Application to the FDA. They also are eligible for accelerated approval and priority review, under which drugs for serious conditions fulfilling an unmet medical need can be approved based on a surrogate endpoint. We are optimistic that GEN-1 represents a game-changer for women with advanced ovarian cancer who have limited treatment options.”

GEN-1 is the subject of Celsion’s Phase II OVATION 2 Study, which combines GEN-1 with standard-of-care neoadjuvant chemotherapy (NACT) in patients newly diagnosed with Stage III/IV ovarian cancer. NACT is designed to shrink the cancer as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo interval debulking surgery, followed by three adjuvant cycles of chemotherapy and up to nine additional weekly GEN-1 treatments, the goal of which is to delay progression and improve overall survival. The OVATION 2 Study is an open-label, 1-to-1 randomized trial, 80% powered to show the equivalent of a 33% improvement in progression-free survival (PFS) (HR=0.75), the primary endpoint, when comparing the treatment arm (standard of care + GEN-1) with the control arm (standard of care alone).

As Celsion has previously announced, it has shared with the FDA data from the Phase I portion of the Phase I/II OVATION 2 Study that showed successful tumor resections, with seven out of eight patients (88%) in the GEN-1 treatment arm having a complete tumor resection (R0), which indicates a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed. The NACT-only treatment arm had an R0 resection rate of 50%.

To Read the Complete Article at GlobeNewswire, Click Here

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, operating like a not-for-profit, 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 high unmet medical need. 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.

Stock image by @vitanovski from Depositphotos

02182021

European Commission Grants Vico Therapeutics Orphan Drug Designation for VO659, an Investigational Therapy for Spinocerebellar Ataxia

Vico Therapeutics, a Leiden, the Netherlands, based biotech company focusing on the development of RNA modulating therapies for rare neurological disorders, today announced that the European Commission (EC) has granted orphan drug designation for VO659, Vico’s investigational antisense oligonucleotide therapy for the treatment of Spinocerebellar Ataxia (SCA). The orphan designation was based on a positive opinion from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP). Vico previously received orphan drug designation for VO659 in Huntington Disease.

“Spinocerebellar Ataxias make up a great part of the so called polyglutamine disorders, debilitating and progressive diseases, leading to significant impairment of mobility, speech, and multiple other daily activities of patients suffering from this condition. So far, there is no treatment which can halt the progression of these diseases,” said Rupert Sandbrink M.D. Ph.D., Chief Medical Officer at Vico.

“Our investigational RNA modulating therapy is designed to lower the mutant protein levels causing these neurodegenerative diseases. We’re pleased to receive orphan drug designation, which recognizes both the unmet medical need for patients living with spinocerebellar ataxias, and the potential of our approach.”

Qualified for orphan drug designation are drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives developing companies certain benefits, including clinical protocol assistance, reduced regulatory fees, research grants and 10 years of market exclusivity following regulatory approval.

About SCA
Spinocerebellar ataxia’s (SCA) are a group of rare, progressive hereditary genetic disorders that affects the cerebellum, brain stem and spinal cord. More than 30 types of SCAs have been identified to date (SCA1–SCA36), and the most common SCAs (types 1, 2, 3, 6 and 7) are caused by translated CAG trinucleotide repeat expansions that encode elongated polyglutamine (polyQ) stretches in the respective disease proteins. Presence of the elongated polyQ stretch confers pathogenic properties to the resulting protein through a dominant gain-of-function mechanism, resulting in degeneration of specific neuronal subpopulations that differ between the different SCA types. For SCA1 and SCA3 disease manifestation includes ataxia of gait, stance, and limbs, dysarthria, and oculomotor abnormalities. To date, there are no disease-modifying therapies.

To Read the Complete Article at B3C Newswire, Click Here

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, operating like a not-for-profit, 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 high unmet medical need. 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.

Stock image by @tatsianama from Depositphotos