Rare Diseases
CEO Matt Gline talked to Scrip about preparing for the launch of brepocitinib in dermatomyositis, lessons learned from Vtama, and how smaller biopharmas are delivering launch successes.
The company licensed worldwide rights to the IBAT inhibitor from GSK just days ahead of the drug’s US FDA approval for cholestatic pruritus associated with primary biliary cholangitis.
With the FDA accepting its refiled BLA and setting an Aug. 22 PDUFA date, Capricor said its cell therapy could offer skeletal and cardiac benefits to Duchenne muscular dystrophy patients.
The company remains blinded to data on functional gains, holding that for future US FDA accelerated approval discussions, but Phase I/II results show microdystrophin production without liver toxicity seen with Sarepta’s Elevidys.
The BLA for the company’s enzyme replacement therapy tividenofusp alfa has an April 5 action date, following on the heels of a complete response letter for Regenxbio’s Hunter syndrome gene therapy.
Shares have surged in companies who have fallen foul of Prasad’s stricter approach, but it is not clear whether a future successor will continue or reverse his policies.
For $2.5bn, the deal will bring Servier a pediatric glioma drug to sell alongside its own Voranigo, and an early-stage clinical pipeline in oncology.
The FDA has reiterated its request for a sham surgery-controlled, Phase III trial of uniQure’s gene therapy candidate for Huntington’s disease, an idea some call unethical.
Ascendis Pharma’s accelerated US approval of once-weekly Yuviwel introduces the first competitor to BioMarin’s Voxzogo in achondroplasia, setting up a dosing-driven competitive dynamic in the CNP segment.
Aardvark’s pause of its Phase III PWS trial after a cardiac signal in a separate study has shaken confidence in the drug’s once‑touted safety edge and has triggered a sharp fall in shares.
The new indication for ocular myasthenia gravis would target about 7,000 patients and provide another growth opportunity for the blockbuster FcRn inhibitor.
The controversial Doug Ingram will depart for personal reasons, following the diagnosis of two family members with myotonic dystrophy.
The company blamed high placebo responses in Latin America and noted greater efficacy in sicker pulmonary arterial hypertension patients that make its kinase inhibitor worthy of FDA filing.
PTC sees the PKU drug approved in July as its near-term growth driver, with geographic expansion expected. It is working with the FDA to refile vatiquinone for Friedreich’s ataxia.
PTC apparently has accepted defeat on trying to gain US approval of its drug for DMD, after 12 years, a complete response and two refuse-to-file letters.
The Danish firm’s Yorvipath produced strong growth during its first full year on market, and Ascendis expects US approval this year for a weekly competitor to BioMarin’s Voxzogo in achondroplasia.
The company’s positive topline Phase III results for the oral drug showed improvements in growth and body proportionality, including gains not seen with standard-of-care injections.
The FDA cited various issues with the CAMPSIITE trial in Hunter syndrome and offered multiple paths forward, but the biotech said that could be a challenge given the rarity of the disease.
The company announced positive Phase II results for the JAK1/TYK2 inhibitor in cutaneous sarcoidosis, which has no approved treatments.
Enthusiastic predictions from industry leaders for ongoing progress in obesity, oncology, immunology, neuroscience and more were supported by optimism for the potential of AI and other technological advances to enable breakthroughs in processes as well as pipelines.