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.
The U.K. medical research charity Lifearc has launched the first part of a £100 million (US$130 million) plan to promote translation of biomedical research into therapies for rare diseases, opening the program with a £2.5 million call for projects to repurpose existing drugs to treat the debilitating inherited skin disease epidermolysis bullosa (EB).
Scientists in the U.K. have reacted with dismay to the announcement that the National Cancer Research Institute (NCRI) is to close, 22 years after it was set up to coordinate the efforts of industry, government research agencies and medical charities that sponsor and fund clinical trials.
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