NEW YORK, Feb. 29, 2024 (GLOBE NEWSWIRE) -- PRISM MarketView, a leading provider of unbiased market insight and company news, recognizes the remarkable contributions of companies developing treatments for the 10,000 rare diseases affecting more than 30 million Americans.
All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders. Approximately 95% of rare diseases have no treatment.
Today on Rare Disease Day, PRISM MarketView highlights emerging companies working to deliver life changing treatments for those living with rare diseases.
Abeona is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. In November 2023, Abeona was granted Priority Review for its Biologics License Application (BLA) for pz-cel (prademagene zamikeracel), for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a severe rare disease characterized by extremely fragile, and extensive blistering and wounds. The company has received a Prescription Drug User Fee Act (PDUFA) target date of May 25, 2024. Abeona’s market cap currently stands at $170.76 million.
Panbela Therapeutics is a clinical-stage biopharmaceutical company developing disruptive therapeutics for urgent unmet medical needs. In April 2023, Panbela regained the worldwide rights to develop and commercialize Flynpovi, a potential treatment for familial adenomatous polyposis (FAP), a rare, inherited cancer. Cancer Prevention Pharmaceuticals, Inc. completed a FAP-310 Phase 3 trial studying the efficacy and safety of the combination of eflornithine and sulindac (Flynpovi), as compared with either drug alone, in adults with FAP. The study showed 100% risk reduction in the need for surgery in patients with an intact lower gastrointestinal anatomy. Panbela has a market cap of $4.243 million.
Voyager is a biotech company leveraging the power of human genetics to modify the course of neurological diseases. The company is targeting the rare disease, Friedreich’s Ataxia (FA), which affects certain nerves in the body and can result in difficulty walking, fatigue, changes in sensation, and slowed speech. This month Voyager selected a lead development candidate in its FA program, triggering a $5 million milestone payment, which the company expects to receive in the first quarter of 2024. Under the terms of Voyager’s 2019 collaboration agreement with Neurocrine Biosciences, Voyager is eligible to receive up to $1.3 billion in potential development and commercial milestone payments, tiered royalties on net sales, and program funding. Voyager has a market cap of $498.091 million.
Capricor is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. This month, the company announced it had been granted an in-person Type-B meeting with the FDA to discuss chemistry, manufacturing and controls (CMC) plans for commercial launch for the potential launch of its treatment for Duchenne Muscular Dystrophy (DMD). DMD is a rare and severe form of inherited muscular dystrophies and leads to progressive muscle fiber degeneration and weakness. Capricor has been granted Regenerative Medicine Advanced Therapy (RMAT) and orphan drug designations for the use of drug candidate, CAP-1002, in DMD. Capricor’s market cap is currently $132.102 million.
Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. In September 2023, the company announced positive topline results from a long term treatment study evaluating DCCR (Diazoxide Choline) extended-release tablets for the treatment of the rare disease, Prader-Willi syndrome (PWS). The company’s shares rose more than 500% following the news.Soleno has been granted FDA and EU Orphan Drug designation for DCCR in the treatment of PWS and FDA Fast Track Designation. Soleno has a market cap of $1.549 billion.
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