May is a busy month for the U.S. Food and Drug Administration (FDA)’s calendar for new drug reviews. Here’s a look.
May is a busy month for the U.S. Food and Drug Administration (FDA)’s calendar for new drug reviews. Here’s a look.
Heron Therapeutics’ HTX-011 for Postoperative Pain
Heron Therapeutics has a target action date of May 12, 2021, for its New Drug Application (NDA) for HTX-011 for postoperative pain. The application was resubmitted on November 13, 2020, after a Type A End-of-Review meeting with the FDA in September 2020, which was held to clarify questions raised in a Complete Response Letter (CRL) the FDA issued in June 2020. The CRL said the agency couldn’t approve the NDA at that time and needed more non-clinical information. There were four non-clinical issues, three related to preclinical reproductive toxicology studies and the fourth tied to a change in the manufacturing release specification of allowable impurity levels based on animal toxicology studies.
HTX-011 is a non-opioid analgesic, a dual-acting, fixed-dose combination of the local anesthetic bupivacaine with a low dose of the nonsteroidal anti-inflammatory meloxicam. In Phase III studies it significantly decreased pain and opioid use through 72 hours compared to bupivacaine solution, the standard-of-care local anesthetic for postoperative pain control.
In September 2020, the European Commission (EC) granted marketing authorization for the drug under the brand name Zynrelef for somatic postoperative pain from small- to medium-sized surgical wounds in adults.
Apellis Pharma’s Pegcetacoplan for PNH
Apellis Pharmaceuticals has a target action date of May 14 for pegcetacoplan for treatment of Paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease that causes red blood cells to break apart (hemolysis). Pegcetacoplan is a targeted, C3 therapy engineered to regulate excessive activation of the complement cascade, part of the immune system. The drug is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b.
On December 10, 2020, the company and Sobi Swedish Orphan Biovitrum AB announced positive topline results at Week 48 of the Phase III Pegasus trial, which showed sustained hematological and clinical improvements in patients with PNH who received pegcetacoplan. The safety profile was also consistent with previous reported data.
At the company’s first-quarter financial report on April 28, 2021, Cedric Francois, co-founder and chief executive officer of Apellis, said, “With a potential U.S. approval for pegcetacoplan just a couple of weeks away, we are at the beginning of a transformational year for Apellis. Our commercial team is prepared to successfully execute our first product launch and meet the needs of PNH patients.”
Sanofi’s Avalglucosidase Alfa for Pompe Disease
Sanofi has a target action date of May 18 for the Biologics License Application (BLA) for avalglucosidase alfa for long-term enzyme replacement therapy for patients with Pompe disease. The drug is designed to improve the delivery of acid alpha-glucosidase (GAA) enzyme to muscle cells. Pompe disease is a deficiency of the enzyme, a rare, degenerative muscle disorder that can impact a patient’s ability to move and breathe.
On February 2, 2021, at the 17th annual WORLDSymposium, the company presented data that supported the BLA. The drug has also been granted Breakthrough Therapy and Fast Track designations by the FDA.
Merck’s Opdivo as Adjuvant Therapy for Esophageal or Gastroesophageal Junction Cancer
Merck has a target action date of May 20 for its supplemental BLA for Opdivo (nivolumab) to treat patients with resected esophageal or gastroesophageal junction (GEJ) cancer in the adjuvant setting, after neoadjuvant chemoradiation therapy (CRT). It was granted Priority Review.
The application was based on data from the Phase III CheckMate -577 trial, which demonstrated positive results in this setting. It met its primary endpoint of disease-free survival (DFS) in patients with esophageal or GEJ cancer after neoadjuvant CRT and tumor resection.
ADC Therapeutics’ Lonca for Diffuse B-Cell Lymphoma
ADC Therapeutics has a target action date of May 21 for its BLA for Lonca (loncastuximab tesirine) for treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL). The application was built on data from LOTIS 2, the pivotal Phase II trial of the drug in this patient population after two or more lines of previous therapy. Lonca is an antibody drug conjugate (ADC) made up of a humanized monoclonal antibody against human CD19 and conjugated via a molecular linker to a pyrrolobenzodiazepine (PBD) dimer cytotoxin. In the trial, the drug demonstrated a 48.3% overall response rate (ORR), which exceeded the target primary endpoint.
The FDA approved it on April 23, 2021 for patients with r/r large C-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from low-grade lymphoma and also high-grade B-cell lymphoma. On May 5, the company announced the drug, which is being marketed as Zynlonta was included in the latest National Comprehensive Cancer Network Clinical Practice Guidelines (NCCN Guidelines) in oncology for B-cell Lymphomas.
“The rapid inclusion of Zynlonta in the NCCN guidelines reinforces our work on behalf of patients who have been heavily pretreated and have difficult-to-treat disease,” said Jay Feingold, senior vice president and chief medical officer of ADC Therapeutics. “Importantly, there is a broad range of pretreated patients needing new therapies, including those who are transplant eligible and ineligible, and patients who previously received stem cell transplant or CAR-T cell therapy.”
