After the initial approvals in monogenic inherited diseases, the scope of gene therapy is widening, with new delivery routes, novel vectors, cell-specific targeting and products aiming to treat chronic disorders, all making headway in 2023.
Drug guidances are still pouring forth from the U.S. FDA as 2023 winds to an end. The latest batch deals with issues as varied as the reformulation of drug products that use carbomers manufactured with benzene, potency assurance for cellular and gene therapies, the quality of topical eye treatments, and the development of drugs and biologics for rare diseases.
On-again, off-again investor enthusiasm for Uniqure NV’s Huntington’s disease (HD) gene therapy AMT-130 got another boost as the company followed this summer’s news from phase I/II trials with additional interim data. Shares of Uniqure (NASDAQ:QURE) closed Dec. 19 at $6.64, down $1.34, or 17%, as the company offered results on up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European experiment.
Both Vertex Pharmaceuticals Inc.’s Casgevy (exagamglogene autotemcel, exa-cel) and Bluebird Bio Inc.’s Lyfgenia (lovotibeglogene autotemcel, lovo-cel) received U.S. FDA approval Dec. 8, providing 16,000 American sickle cell patients who have recurring vaso-occlusive events with access to the first cell-based gene therapies.
With the number of high-priced cell and gene therapies expected to grow dramatically over the next decade, U.S. Sen. Bill Cassidy (R-La.) is asking for input from patients, doctors, manufacturers, payers and policy wonks on how to pay for the treatments and ensure patient access when they become more mainstream across the ultra-rare disease spectrum.
Arriving on the gene therapy scene with an undisclosed seed funding sum, Alveogene is tackling respiratory diseases with high unmet need via a next-generation lentiviral delivery platform to advance into the clinic a candidate for rare inherited disorder alpha-1 antitrypsin deficiency.
The latest firm to brave the rough IPO market, Lexeo Therapeutics Inc. made its Nasdaq debut after pricing about 9.1 million shares at $11 per share, raising proceeds of $100 million to advance its early clinical work on gene therapies for cardiovascular and neurological diseases.
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.
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.
Failing to meet the primary endpoint in its confirmatory phase III Embark trial, Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec), which received accelerated approval in June and was priced at $3.2 million, has one of three fates in its future, all of which are dependent on how the U.S. FDA perceives the data. Based on secondary endpoints showing statistical significance and a recent positive meeting with the agency, Sarepta could continue to market Elevidys under its current label for 4- and 5-year-old ambulatory Duchenne muscular dystrophy (DMD) patients; Sarepta is filing the postmarketing requirement needed to transition from accelerated to full approval.
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