With a landmark U.K. approval in hand for Casgevy (exagamglogene autotemcel [exa-cel]) to treat sickle cell disease and transfusion-dependent beta thalassemia, Crispr Therapeutics AG and partner Vertex Therapeutics Inc. are turning their attention to the PDUFA dates set by the U.S. FDA for the treatment in both conditions.
With the U.S. FDA giving the green light to Takeda Pharmaceutical Co. Ltd.’s Adzynma for treating a rare blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme, the company has won two approvals in two days after the FDA approved fruquintinib a day earlier.
Among the avalanche of abstracts for the upcoming American Society of Hematology (ASH) annual conference released early on Nov. 2 were data from Arcellx Inc.’s phase I study of CART-ddBCMA in patients with relapsed or refractory multiple myeloma. The results caught investors’ attention as the company’s stock (NASDAQ:ACLX) jumped 18.5% upward to close at $40.77 per share.
Had it been asked to, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee would have voted Oct. 31 to recommend approval of Vertex Pharmaceutical Inc. and Crispr Therapeutics AG’s exagamglogene autotemcel, or exa-cel, as a one-time transformative treatment for severe sickle cell disease in individuals 12 and older.
It’s just a discussion next week of Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG’s sickle cell disease (SCD) therapy, with no vote expected from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee. The FDA isn’t questioning the drug’s efficacy as much as it is concerned about the effects of off-target editing.
Five years after Gilead Sciences Inc. gave up on momelotinib in the wake of two phase III failures in myelofibrosis, the JAK1/2 and ACVR1 inhibitor has found its way to the market in the hands of GSK plc. Branded Ojjaara, the drug gained U.S. FDA approval for use in intermediate- or high-risk myelofibrosis patients with anemia regardless of prior administration with JAK inhibitors such as Jakafi (ruxolitinib, Incyte Corp.).
Star Therapeutics Inc. brought on board six new investors in its oversubscribed $90 million series C round, raising the company’s total funding to more than $190 million since its founding in 2018 to advance in-house drug discovery efforts via formation of portfolio companies. Since emerging from stealth in early 2022, Star has launched two aptly named companies, Electra Therapeutics Inc. and Vega Therapeutics Inc., with plans to unveil additional ventures arising from its antibody discovery efforts.
The U.S. FDA has awarded Bristol Myers Squibb Co. (BMS) with its third approval for treating anemia with Reblozyl (luspatercept-aamt). Specifically, the approval is for treating anemia without previous erythropoiesis stimulating agent use in adults with very low- to intermediate-risk myelodysplastic syndromes who may require regular red blood cell transfusions.
Shares of Fulcrum Therapeutics Inc. shot up 38.5% on Aug. 22 following news that the U.S. FDA had lifted the clinical hold on the company’s phase Ib sickle cell disease candidate, FTX-6058.
Hutchmed (China) Ltd.’s sovleplenib (HMPL-523) met the primary endpoint of durable response rate and all secondary endpoints in the pivotal phase III trial in adults with primary immune thrombocytopenia (ITP) in China.
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