The U.S. development path for rare disease treatments is strewn with numerous challenges, not least of which are the regulatory hurdles. For companies developing promising candidates to treat ultra-rare diseases and the patients who are running out of time, the regulatory obstacles in the U.S. may seem almost insurmountable. And new concerns about drug development in general could make those barriers even higher.
The U.S. FDA marked the 40th anniversary of the Orphan Drug Act with Rare Disease Day 2023 as Robert Califf, the agency’s commissioner of food and drugs, opened the day by expressing his wonder and accompanying concern regarding gene editing and gene therapy.
Taysha Gene Therapies Inc.’s stock (NASDAQ:TSHA) struggled Feb. 1 following the U.S. FDA’s recommendation that the company dose more patients in a double-blind, placebo-controlled study of TSHA-120 for the ultra-rare indication giant axonal neuropathy.
The successful suitor for Horizon Therapeutics plc has been revealed as Amgen Inc., which will buy the Dublin-based company for $27.8 billion in cash in what is by far the year’s biggest acquisition. The deal brings successful biologics into Amgen’s portfolio that include Tepezza (teprotumumab), a monoclonal antibody targeting insulin-like growth factor 1 for treating thyroid eye disease, Krystexxa (pegloticase), a pegylated uricase enzyme for treating chronic refractory gout, and Uplizna (inebilizumab), a monoclonal antibody targeting insulin-like growth factor 1 for treating thyroid eye disease.
Bloomsbury Genetic Therapies Ltd. has raised £5 million (US$5.5 million) in a seed round, to take four gene therapy programs based on research carried out by the scientific founders at University College London into clinical development.
After winning the backing of European regulators, Biomarin Pharmaceutical Inc. is returning to the U.S. FDA with its hemophilia A gene therapy, valoctocogene roxaparvovec, following a rejection in August 2020. It's armed with data it hopes will assuage concerns about long-term safety and benefits.
Versantis AG has agreed terms with Genfit SA in an acquisition deal that involves CHF40 million (US$41.4 million) up front, plus up to CHF65 million in milestones, and one third of the proceeds from a potential sale of a rare pediatric disease priority review voucher, should the U.S. FDA grant one.
Access to advanced therapies proved to be a major talking point at a conference in London, following the U.S. approval of Bluebird Bio Inc.’s Zynteglo (betibeglogene autotemcel) cell-based gene therapy for beta thalassemia and its $2.8 million price tag. Regulators in Europe backed Zynteglo in 2019 but Bluebird opted to withdraw the therapy in 2021 after deciding that the complex thicket of pricing bodies in Europe was too difficult to negotiate.
Apollo Therapeutics Group Ltd. has bought worldwide rights to Avalo Therapeutics Inc.’s anti-IL-18 antibody, camoteskimab, in a deal worth up $89 million, taking on development of the early stage drug for arthritis and potentially other diseases.
Sarepta Therapeutics Inc. said it plans to file a BLA for its gene therapy for Duchenne muscular dystrophy (DMD), SRP-9001, with the U.S. FDA, potentially setting up a decision in the first half of 2023 for the therapy developed in partnership with Switzerland’s Roche Holding AG. The Cambridge, Mass.-based biotech said the BLA will seek accelerated approval for the therapy, also known as delandistrogene moxeparvovec, for ambulant individuals with DMD.
