FDA Action Alert: Sanofi, Motif Bio and Merck

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Over the next two weeks, the U.S. Food and Drug Administration (FDA) has three upcoming decisions. Here’s a look.

Sanofi’s Biologics License Application for Caplacizumab for aTTP

France’s Sanofi has a target action date of February 6 for its caplacizumab for acquired thrombotic thrombocytopenic purpura (aTTP), a life-threatening, autoimmune-based blood clotting disorder. The disease is marked by extensive blood clot formation in the small blood vessels of the body. This can lead to severe thrombocytopenia, which is a very low platelet count, microangiopathic hemolytic anemia, which is red blood cell destruction, and restricted blood supply throughout the body, or ischemia. This can cause widespread organ damage, particularly in the heart and brain.

The drug was approved under the brand name Cablivi by the European Commission in August 2018. The drug was developed by Ablynx, a Sanofi company. It is the company’s first Nanobody-based drug to receive approval and the first newly approved product that is part of Sanofi Genzyme’s Rare Blood Disorders franchise.

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Motif Bio’s Antibiotic for Skin Infections

Motif Bio, with offices in New York and London, has a target action date of February 13 for its New Drug Application (NDA) for iclaprim, a targeted, Gram-positive investigational antibiotic for the treatment of acute bacterial skin and skin structure infections. The company also plans to develop the antibiotic for hospital-acquired pneumonia (HABP), including ventilator-associated bacterial pneumonia (VABP). A Phase II trial in patients with HABP has been completed and a Phase III trial is planned.

Iclaprim has received Qualified Infectious Disease Product (QIDP) designation from the FDA along with Fast Track designation for the ABSSSI indication.

The company presented iclaprim data in October at the IDWeek conference in San Francisco, showing it was non-inferior to vancomycin in a pooled analysis of a subgroup of patients in the REVIVE Phase III clinical trials in patients with wound infections.

G. Ralph Corey, Gary Hock Professor at Duke University School of Medicine and principal investigator of the REVIVE-2 trial, stated at the time, “Wound infections, including surgical site infections, can be difficult to treat and it was important to see that iclaprim was non-inferior to standard of care in treating these types of infections. Additionally, in the iclaprim treatment arm, there was no evidence of nephrotoxicity, while two patients in the vancomycin arm demonstrated high serum creatinine levels.”

Merck’s Checkpoint Inhibitor Keytruda Up for Another Indication

Merck & Company has a target action date of February 16 for its supplemental Biologics License Application (sBLA) for Keytruda, its anti-PD-1 therapy. It is being reviewed as adjuvant therapy in patients with resected, high-risk stage III melanoma. The sBLA is based on data from the pivotal Phase III EORTC1325/KEYNOTE-054 trial, which showed significant benefit in recurrence-free survival. The data were presented at the American Association for Cancer Research (AACR) Annual Meeting 2018 and published in The New England Journal of Medicine.

In June, when the acceptance of the sBLA was announced, Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories, stated, “EORTC1325/KEYNOTE-054 was the first trial with Keytruda to demonstrate a recurrence-free survival benefit in the adjuvant setting, and we continue to actively investigate Keytruda in the adjuvant or neoadjuvant setting across our broad clinical development program.”

On Jan. 3, Merck announced that Keytruda had been approved for this indication by the Japan Pharmaceuticals and Medical Devices Agency (PMDA), as well as for four other indications: in combination with pemetrexed and platinum-based chemo for first-line unresectable, advanced/recurrent nonsquamous NSCLC regardless of PD-L1 expression; in combination with carboplatin and paclitaxel or nab-paclitaxel for first-line treatment of unresectable, advanced/recurrent squamous NSCLC regardless of PD-L1 expression; monotherapy in first-line treatment of PD-L1-positive unresectable, advanced/recurrent NSCLC; and as monotherapy for advanced/recurrent MSI-H solid tumors that have progressed after chemotherapy.

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