FDA Action Alert

 

Wrapping up June and ahead of the July 4 holiday, the U.S. Food and Drug Administration (FDA) has a busy two-week period coming up for drug approvals. Here’s a look.

Karyopharm’s Xpovio for B-Cell Lymphoma

Newton, Massachusetts-based Karyopharm Therapeutics has a target action date of June 23 for its supplemental New Drug Application (sNDA) for oral Xpovio (selinexor) for adults with relapsed or refractory diffuse large  B-cell lymphoma (RR DLBCL) who have received at least two previous therapies. Xpovio is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. It was approved in July 2019 in combination with dexamethasone for adults with relapsed refractory multiple myeloma (RRMM) who have received at least four previous therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody.

Zogenix’s Fintepla for Seizures Associated with Dravet Syndrome

Zogenix has a target action date of June 25 for its NDA for Fintepla (fenfluramine oral solution) for seizures associated with Dravet syndrome. This is a three-month extension from its previous NDA. The extension allowed the FDA time to review additional data the company submitted in response to an information request from the agency. The NDA was originally accepted in November 2019 under the agency’s Priority Review program. It was based on data from two positive pivotal Phase III trials in Dravet and an interim analysis from an ongoing open-label extension trial, which included 232 patients treated for up to 21 months.

Chiasma’s Mycapssa for Acromegaly

Needham, Massachusetts-based Chiasma had a target action date of June 26 for its NDA resubmission for its Mycapssa for maintenance treatment of adults with acromegaly. Acromegaly usually develops when a benign tumor of the pituitary gland generates too much growth hormone. This leads to significant health problems. Common features are facial changes, intense headaches, joint pain, impaired vision and enlargement of the hands, feet, tongue and internal organism.

The company reported positive topline results from its Phase III CHIASMA OPTIMAL trial in July 2019, with 58% of patients on the drug maintaining their IGF-1 response compared to 19% of the placebo group.

Heron Therapeutics’ HTX-011 for Post-Operative Pain

San Diego-based Heron Therapeutics has a target action date of June 26 for its resubmission of its NDA for HTX-011 for postoperative pain. HTX-011 is a dual-acting, fixed-dose combination of the local anesthetic bupivacaine with a low dose of the nonsteroidal anti-inflammatory drug meloxicam. The company received a Complete Response Letter (CRL) from the FDA on April 30, 2019, relating to chemistry, manufacturing and controls and non-clinical information. The company had been reinspected by the FDA with no Form 483 citations observed and the site was approved. There were no efficacy issues.

On June 4, the company published the results of Study 209, a Phase IIb trial of HTX-011 in primary unilateral total knee arthroplasty (TKA). All primary and key secondary endpoints were achieved, with the drug showing statistically significant decreases in pain post-surgery.

Intercept Pharma’s Ocaliva for in Liver Fibrosis Due to NASH

Intercept Pharmaceuticals, based in New York, has a target action date of June 26 for its NDA for Ocaliva (obeticholic acid, OCA) in liver fibrosis due to NASH. Nonalcoholic steatohepatitis (NASH) is a fatty liver disease similar to cirrhosis of the liver but occurs in people with little or no alcohol use. Ocaliva is indicated for primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (DCA) in adults with inadequate response to UDCA or as a monotherapy in patients unable to tolerate UDCA.

Merck’s Keytruda for R/R Cutaneous Squamous Cell Carcinoma

Merck’s blockbuster checkpoint inhibitor Keytruda (pembrolizumab) has a target action date of June 29. The supplemental Biologics License Application (sBLA) is for recurrent and/or metastatic cutaneous squamous cell carcinoma (cSCC) that can’t be cured by surgery or radiation.

Keytruda is an anti-PD-1 checkpoint inhibitor that is involved in more than 900 clinical trials across a wide variety of cancers and treatment settings. It has been approved for numerous indications, including melanoma, lung cancer, head and neck cancer, classical Hodgkin Lymphoma and others.