Reneo Pharmaceuticals Inc. is halting development of its only asset, mavodelpar, after a phase IIb study missed its primary and secondary efficacy endpoints. The selective peroxisome proliferator-activated receptor (PPAR)-delta agonist was being developed to treat to rare genetic mitochondrial diseases.
The U.S. FDA has approved Novartis AG’s Fabhalta (iptacopan) as the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria, a rare blood disease that impairs blood cell production. This is the only factor B inhibitor of the immune system’s complement pathway and is expected to be on the market before December ends. Fabhalta has plenty of competition from already-approved therapies and more treatments are in development.
Astrazeneca plc is making a $220 million equity investment and tossing in $25 million up front to Cellectis SA as part of a new collaboration agreement. The deal is part of Astrazeneca’s efforts, including a July licensing agreement worth about $1 billion with Pfizer Inc., to delve deeper into gene therapy for treating cancer and rare diseases.
In a show of bipartisan solidarity, members of the U.S. Senate Special Committee on Aging voiced their support Oct. 26 for a new regulatory pathway to quicken access to new drugs for rare diseases that have no approved treatments.
Japanese specialty global pharma Kyowa Kirin Co. Ltd. has agreed to buy out Orchard Therapeutics plc in a $387.4 million cash takeover that could jump $90 million to reach $477.6 million, contingent on the pending U.S. FDA approval of its EU-approved gene therapy, Libmeldy (atidarsagene autotemcel).
Japanese specialty global pharma Kyowa Kirin Co. Ltd. has agreed to buy out Orchard Therapeutics plc in a $387.4 million cash takeover that could jump $90 million to reach $477.6 million, contingent on the pending U.S. FDA approval of its EU-approved gene therapy, Libmeldy (atidarsagene autotemcel).
The US FDA is offering sponsors of certain drugs and biologics more agency access as part of a pilot program that will be launching in January 2024 with the mission of accelerating the development of new therapies for rare diseases.
Positive top-line phase III study results for olezarsen in treating familial chylomicronemia syndrome has Ionis Pharmaceuticals Inc. looking down the road to U.S. FDA approval. It’s the company’s second attempt at getting an approval for treating the rare disorder.
While the U.S. FDA didn’t ask for more study data or have safety or efficacy concerns, it does want modifications to Alexion, Astrazeneca Rare Disease’s sBLA for Ultomiris (ravulizumab-cwvz) to treat adults with the rare central nervous system disease neuromyelitis optica spectrum disorder. The agency has issued a complete response letter (CRL) requesting changes to Ultomiris’ Risk Evaluation and Mitigation Strategy (REMS) to better validate patients’ meningococcal vaccination status or prophylactic administration of antibiotics before being treated.
South Korean biopharma Connext Co. Ltd. is set on outdoing standard-of-care therapies for Dupuytren’s contracture. The Daegu-headquartered Connext recently secured U.S. FDA IND approval for a phase I/II trial on its recombinant collagenase clostridium histolyticum, called CNT-201, inching closer to its goal of providing an affordable but high-quality therapeutic option for patients with the rare, progressive connective tissue disorder.
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