The U.K. medical research charity Lifearc has launched the first part of a £100 million (US$130 million) plan to promote translation of biomedical research into therapies for rare diseases, opening the program with a £2.5 million call for projects to repurpose existing drugs to treat the debilitating inherited skin disease epidermolysis bullosa (EB).
Scientists in the U.K. have reacted with dismay to the announcement that the National Cancer Research Institute (NCRI) is to close, 22 years after it was set up to coordinate the efforts of industry, government research agencies and medical charities that sponsor and fund clinical trials.
Right on schedule the U.S. FDA gave its blessing for Krystal Biotech Inc.’s topical gene therapy, Vyjuvek (beremagene geperpavec, or B-VEC), an orphan drug, to become the first approved treatment for the rare skin condition dystrophic epidermolysis bullosa (DEB).
On the heels of the marketing OK in Europe, Protalix Biotherapeutics Inc. and the Chiesi Group’s global rare diseases unit scored approval of Elfabrio (pegunigalsidase alfa-iwxj) from the U.S. FDA for adults with Fabry disease.
The dominoes are falling on European clinical trials as the unintended consequences of the EU’s In Vitro Diagnostics Regulation take hold. Last year’s implementation of the regulation has resulted in the delay of up to 160 drug trials to date, with as many as 420 trials expected to be delayed over the next three years, according to an EFPIA member survey.
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.
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