SP-101, an investigational inhaled gene therapy for cystic fibrosis, shows potential in animal study

Treatment with SP-101, an investigational gene therapy that Spirovant Sciences is developing for people with cystic fibrosis (CF) who are unable to benefit from current treatments, effectively increased CFTR gene activity in a ferret model of the disease, the company reported. Katherine Excoffon, PhD, Spirovant’s vice president of research, presented the data at the 2021 North American Cystic Fibrosis Conference (NACFC). Her

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FDA approves new breakthrough therapy for cystic fibrosis

Treatment approved for approximately 90% of patients with cystic fibrosis, many of whom had no approved therapeutic options Source FDA The U.S. Food and Drug Administration today approved Trikafta (elexacaftor/ivacaftor/tezacaftor), the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation. Trikafta is approved for patients 12 years and older

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Thirty patient deaths tied to cystic fibrosis drug following FDA database of side effects

Thirty patients have died after taking Symdeko, a Vertex Pharmaceuticals treatment for cystic fibrosis, according to a government database. The deaths, reported to the Food and Drug Administration’s database of side effects, are tied to Symdeko, a Vertex medicine approved last year. Reports to the database are commonly submitted by health care professionals and often contain

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