Approvals & Launches

The FDA and Sarepta have had a controversial history with the accelerated approvals of the firm’s earlier drugs for Duchenne muscular dystrophy – but the company’s investigational gene therapy, SRP-9001, now under a priority review, could be a genuine breakthrough.

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

Rezlidhia, which was only recently licensed by Rigal from Novo Nordisk-owned Forma Therapeutics, should challenge the market dominance of Servier's Tibsovo in the IDH-1 inhibitor subset of acute myeloid leukemia patients. 

The microbiome therapy won approval for preventing recurrence of Clostridioides difficile infections, but an analyst said the future could be in more targeted approaches.

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

Shionogi’s once-daily oral COVID-19 antiviral finally gets Japan approval after being considered for the third time by an expert panel, paving the way for public availability of the country’s first such home-grown option in early December.

Skyrizi is the first specific IL-23 inhibitor for the treatment of Crohn's disease in Europe, though the drug was approved with a broader label in the US.

The firms’ gene therapy Hemgenix has won a US thumbs up for the treatment of hemophilia B and plans are underway for a US launch with a big price tag that experts agree should pay off in the long-term.

The Belgian group was stunned by an FDA rejection in May for its dual IL-17A and IL-17F inhibitor and although the refiling is complete, UCB may still have to wait six months to get Bimzelx into the all-important US psoriasis market.

Blenrep’s withdrawal will add to GSK’s blockbuster drought, and hopes of separate trial success for the BCMA-targeting drug in earlier stage myeloma may now be harder to achieve.

At nearly $194,000, the wholesale acquisition cost for Tzield came in above analysts’ $100,000-$115,000 estimates, but payer pushback is not expected as the drug faces no competition.

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.