Kisunla for Alzheimer’s, Sirturo for TB, Kar-XT for Schizophrenia, MDMA-based Therapy for PTSD, Seladelpar for PBC, TransCon PTH for Hypoparathyroidism, Afami-Cel for Synovial Sarcoma

New FDA Approvals

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- The FDA has approved donanemab (Kisunla) for early symptomatic Alzheimer's disease, making it the third drug in a new class aimed at slowing cognitive decline in early-stage patients. Eli Lilly will make the drug available within weeks following this approval. Donanemab is an anti-amyloid monoclonal antibody that targets amyloid plaques in the brain, a key feature of Alzheimer's.

- The FDA approval was based on clinical trials showing significant benefits in slowing cognitive decline despite concerns about long-term safety. The advisory committee voted unanimously in favor of the drug, with the FDA granting the approval to Eli Lilly.

- The FDA has approved bedaquiline (Sirturo) for treating multidrug-resistant pulmonary tuberculosis (TB) in adults and children aged five and above. Sirturo initially received accelerated FDA clearance for adults in 2012, with later label expansions for younger patients. Bedaquiline, the first TB drug with a new action mechanism in over 40 years, inhibits mycobacterial ATP synthase, essential for energy production in TB bacteria..

- The FDA is nearing a decision on KarXT, a novel antipsychotic for schizophrenia developed by Karuna Therapeutics. KarXT has shown efficacy in reducing symptom severity in clinical trials, with a favorable side effect profile compared to older antipsychotics. Concerns about insurance coverage exist due to the availability of cheaper generics, but a decision is expected by September 26.

- MDMA-assisted therapy for PTSD, developed by Lykos Therapeutics, has shown significant symptom reduction in clinical trials but faces approval challenges. Despite positive trial results, the FDA advisory panel voted against recommending the drug, citing safety concerns. The FDA's final decision is expected by August 11.

- Seladelpar, under consideration for primary biliary cholangitis (PBC), is a drug Gilead recently acquired through the purchase of CymaBay. If approved by August 14, it would expand Gilead's liver disease portfolio. Clinical trials have shown seladelpar to be effective in reducing the itching associated with PBC.

- TransCon PTH by Ascendis Pharma is a treatment for hypoparathyroidism, designed to replace parathyroid hormone and help patients achieve normal calcium levels. Despite manufacturing concerns delaying the FDA's decision, the drug has already been approved in Europe and the UK. A final FDA decision is expected by August 14.

- Afami-cel, a T-cell receptor therapy for synovial sarcoma developed by Adaptimmune Therapeutics, awaits FDA approval by August 4. The therapy targets the MAGE A4 cancer target and is designed as a single-dose treatment. Clinical trial data supporting the application showed efficacy in treating advanced synovial sarcoma.