A new self-injectable therapy for polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) will be available in January 2024 now that the U.S. FDA has approved Ionis Pharmaceuticals Inc.’s Wainua (eplontersen), a ligand-conjugated antisense oligonucleotide.
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.
How flexible should the U.S. FDA evidentiary standards be for a therapy addressing a significant unmet need in a disease such as amyotrophic lateral sclerosis (ALS)? That’s the question the agency’s Cellular, Tissue and Gene Therapies Advisory Committee will ponder Sept. 27 as it looks at the data for Brainstorm Cell Therapuetics Inc.’s Nurown (debamestrocel), a mesenchymal stromal cell therapy targeting ALS. Nurown is going into the adcom with a bit of a checkered history that includes a refuse-to-file letter and a single phase III trial that failed to demonstrate efficacy for the primary endpoint and all key secondary efficacy endpoints, according to the FDA briefing document.
Shares in Uniqure NV fell sharply on inconclusive interim data from a U.S. phase I/II trial of its gene therapy for Huntington’s disease. The stock (NASDAQ:QURE) hit a six-year low during trading on June 21, ending the day at $11.62, down 40%.
Despite the approval of Biogen Inc.’s Qalsody (tofersen) for treating amyotrophic lateral sclerosis (ALS), there are still hurdles for the drug to clear, including a confirmatory study and setting a price. Because the U.S. FDA granted Qalsody accelerated, not full approval, there are plenty more data to collect in the ongoing confirmatory phase III Atlas study of those who develop ALS symptoms during the trial compared to placebo. The randomized, double-blind, placebo-controlled study is being conducted with those carrying the superoxide dismutase 1 gene genetic mutation but are symptom free.
A twisted, uncertain path has led to a U.S. FDA approval for Biogen Inc.’s Qalsody (tofersen), the first drug targeting a genetic cause of amyotrophic lateral sclerosis (ALS). Qalsody is for ALS associated with a mutation in the superoxide dismutase 1 (SOD1) gene. Patients with SOD-1 mutations account for 2% of ALS cases.
Ionis Pharmaceuticals Inc. and Astrazeneca plc’s candidate for hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN), eplontersen, has continued to improve disease progression out to 66 weeks, according to Ionis.
Despite a failed phase III study, the U.S. FDA suggests in briefing documents that tofersen (BIIB-067) is effective for treating the rare, genetic disease superoxide dismutase 1 amyotrophic lateral sclerosis (ALS). The intrathecally injected therapy is being developing by Biogen Inc. and Ionis Pharmaceuticals Inc. and is at the heart of a March 22 meeting of the agency’s Peripheral and Central Nervous System Drugs Advisory Committee (adcom).
Once a rarity, billion-dollar deals are now coming fast and furious. Now three have appeared in only a few days. Gene therapy developer Voyager Therapeutics Inc. and Neurocrine Biosciences Inc. continued a longstanding partnership by agreeing to develop treatments for neurological diseases and for three new programs with rare CNS targets. Cytomx Therapeutics Inc. and Moderna Inc. entered a deal worth a potential $1.24 billion to Cytomx for generating and developing treatments in oncology and non-oncology. In the third deal, Royalty Pharma plc acquired an interest in Ionis Pharmaceuticals Inc.'s royalty in Biogen Inc.'s Spinraza (nusinersen) and Novartis AG's pelacarsen for up to $1.125 billion.
