FDA Action Alert: Merck, Agios and AMAG Pharma


June is turning out to be a busy month for approvals for the U.S. Food and Drug Administration (FDA). The agency has a slate of PDUFA dates this week, including two for Merck alone. Here’s a look.

Merck has a target action date of June 17 for its new supplemental Biologics License Application (sBLA) for Keytruda as a monotherapy for advanced small cell lung cancer (SCLC) whose disease has progressed after two or more lines of prior therapy. The submission was based on data from the SCLC cohorts of the Phase II KEYNOTE-158 and Phase Ib KEYNOTE-028 clinical trials.

Merck also has a target action date of June 20 for its sBLA for Keytruda, this time in combination with axitinib for the first-line treatment of advanced renal cell carcinoma (RCC). Axitinib (Inlyta) is a tyrosine kinase inhibitor marketed by Pfizer. The submission was based on results from the Phase III KEYNOTE-426 trial. The FDA granted it priority review for this indication.

Click to browse jobs in the life sciences

It can be a little difficult to track all the approvals for Keytruda, Merck’s anti-PD-1 checkpoint inhibitor. The drug is involved in more than 1,000 clinical trials as a monotherapy or in combinations with other drugs. On June 11, for example, Keytruda was approved as a monotherapy in patients whose tumors express PD-L1 or in combination with platinum and fluorouracil (FU), a chemotherapy agent, for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC). This was based on results from the Phase III KEYNOTE-048 trial.

“This approval is a very exciting milestone in the treatment of head and neck cancer and has the potential to transform the way we treat patients with this debilitating disease by offering important new therapeutic options,” stated Barbara Burtness, professor of medicine, Yale School of Medicine and co-director, Development Therapeutics Research Program, Yale Cancer Center. “Metastatic or recurrent head and neck cancer have been an area of significant unmet need, so it is encouraging to have immunotherapy regimens available for patients in the first-line setting.”

Agios Pharmaceuticals has a target action date of June 21 for its supplemental New Drug Application (sNDA) for Tibsovo (ivosidenib) for newly diagnosed acute myeloid leukemia (AML) with an isocitrate dehydrogenase 1 (IDH1) mutation who are not eligible for standard therapy. This is under Priority Review and was accepted under the FDA’s Real-Time Oncology Review pilot program.

Tibsovo is a first-in-class, oral, targeted inhibitor of mutant IDH1. The sNDA is based on data from untreated AML patients in the Phase I dose-escalation and expansion trial of ivosidenib in patients with newly diagnosed AML ineligible for standard treatment.

AMAG Pharmaceuticals has a June 23 target action date for bremelanotide for Hypoactive Sexual Desire Disorder (HSDD). HSDD is noted by low sexual desire and associated distress. It is believed to be caused by an imbalance in brain chemicals that affect sexual excitation or inhibition. The drug is designed to be used ahead of a sexual encounter. It has been evaluated in more than 30 clinical trials with more than 2,500 women.

The New Drug Application was built on data from two large double-blind placebo-controlled Phase III trials where the drug met the pre-specified co-primary efficacy endpoints of improvement in desire and decrease in distress linked to low sexual desired as measured by validated patient-reported outcomes.

The women in the study were given the option of participating in an open-label safety extension trial for another 12 months. Almost 80% chose to participate, all receiving bremelanotide.

AMAG licensed the drug, which has a brand name of Vyleesi, from Palatin Technologies.

Back to news