The EMA has been sent back to the drawing board to re-evaluate PTC Therapeutics Inc.’s Duchenne muscular dystrophy therapy Translarna (ataluren), after failing to get the usual rubber stamp following its recommendation in January that the drug’s conditional approval be withdrawn.
Ultragenyx Pharmaceutical Inc. anticipates a meeting later this year with the U.S. FDA to discuss a BLA filing for gene therapy DTX-401 as the first potential medical treatment for glycogen storage disease type 1a (GSD1a) after the phase III study hit its primary endpoint and two key secondary endpoints.
A boy participating in the phase II Daylight study of Duchenne muscular dystrophy (DMD) “has passed away suddenly,” according to Pfizer Inc. The participant had received fordadistrogene movaparvovec, a mini-dystrophin gene therapy, in early 2023. The fatal serious adverse event was reported May 3 as a cardiac arrest, Pfizer told BioWorld. Pfizer, together with the independent external data monitoring committee, is reviewing the data to understand the potential cause, the company added.
Philadelphia-based Latus Bio Inc., co-founded by serial biotech entrepreneurs P. Peter Ghoroghchian and Beverly Davidson, launched on May 2 with two lead adeno-associated virus (AAV)-based gene therapy candidates and $54 million in a series A financing.
Ending a late 2021 deal that was potentially worth billions, Moderna Inc. and Metagenomi Inc. are going their separate ways. The two had been collaborating on gene-editing R&D to develop therapies for treating serious genetic diseases. Moderna said it agreed with Metagenomi to end the deal as “Moderna continues to strategically prioritize its research and development investments.”
Pfizer Inc.’s Beqvez (fidanacogene elaparvovec) won FDA approval for use in adults with hemophilia B, making it the second adeno-associated viral (AAV) vector-based gene therapy available for patients in the U.S., following the late 2022 approval of CSL Behring’s Hemgenix (etranacogene dezaparvovec).
Seven years after embarking on in vivo therapeutic development using CRISPR/Cas gene-editing technology with Intellia Therapeutics Inc., Regeneron Pharmaceuticals Inc. is bringing another company into the collaborative fold. Regeneron will pay Mammoth Biosciences Inc. $100 million, including $95 million as an equity investment, and an up-front payment. Mammoth also could bring in up to $370 million for each target in milestones along with royalties on net sales from products created through the collaboration.
Led by executives who helmed The Medicines Co., Metsera Inc. has launched with $290 million in financing to develop drugs for treating weight loss, obesity-related conditions and metabolic diseases. The company is stepping into high profile and lucrative glucagon-like peptide-1 territory forged by Eli Lilly and Co.’s Zepbound (tirzepatide) for chronic weight management and Novo Nordisk A/S’s Wegovy (semaglutide), also approved for long-term weight management.
Some gene therapies could be big winners under the changes the U.S. Centers for Medicare & Medicaid Services (CMS) is proposing to Medicare’s new technology add-on program (NTAP) for its fiscal 2025 inpatient prospective payment system.
Two days after the U.S. FDA announced approval of gene therapy Lenmeldy (atidarsagene autotemcel), making it the first treatment option for rare disease metachromatic leukodystrophy, Orchard Therapeutics and parent firm Kyowa Kirin Co. Ltd. disclosed the wholesale acquisition price of $4.25 million for the one-time treatment, which edges out hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) to become the world’s most expensive drug.
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