FDA Actions in Brief

Scemblix (asciminib) is a new first-line option for adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP).

Regulators are also reviewing Teva’s application for its Prolia biosimilar candidate. The FDA’s decisions are expected mid-year 2025.

PTC Therapeutics' NDA resubmission of Translarna was based on data from Study 041 and the long-term STRIDE registry. No PDUFA action date was provided for regulators.

Prademagene zamikeracel (pz-cel) could be a new treatment for rare genetic disease recessive dystrophic epidermolysis bullosa (RDEB).

Qsymia’s manufacturer also released postmarketing data showing the oral therapy for weight loss was associated with reductions in 24-hour mean systolic blood pressure.

Gavreto used to treat metastatic RET fusion-positive non-small cell lung cancer and RET fusion-positive thyroid cancer.