FDA Approvals Roundup: Inmazeb, Wakix, Venclexta
A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).New approvals
Inmazeb approved as first treatment for Ebola virus
Regeneron’s Inmazeb (atoltivimab, maftivimab, and odesivimab-ebgn combination) has been approved as the first treatment for Zaire ebolavirus (Ebola virus) infection in adults and children. The therapy received orphan drug and breakthrough therapy designations for treating the infection.
The potentially fatal disease is transmitted through direct contact with blood, body fluids, and tissues of infected people or animals, as well as surfaces or materials contaminated with the fluids. The therapy, a combination of the three monoclonal antibodies (mAbs), binds with the infection-promoting glycoprotein on the virus surface, preventing the virus from entering the cell and infecting the individual.
Inmazeb’s approval followed the December 2019 approval of the first Ebola virus vaccine, based on findings from a study done during the 2014-2016 Ebola outbreak in Guinea.
The drug’s safety and efficacy were evaluated in the multicenter, open-label, randomized, controlled PALM trial and an expanded access international program during an outbreak of the Ebola virus in the Democratic Republic of the Congo in 2018-2019. In the PALM trial, 382 patients received a single intravenous infusion of Inmazeb (50 mg of each mAb) or an investigational control. After 28 days, 33.8% of patients receiving Inmazeb had died, compared with 51% of those receiving a control. An additional 228 patients received Inmazeb during the expanded access program.
“Today’s approval highlights the importance of international collaboration in the fight against Ebola virus,” said John Farley, MD, MPH, director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research in a statement.
New indications
Wakix gets expanded indication for adults with narcolepsy-related cataplexy
Harmony Biosciences’ Wakix (pitolisant tablet) has received an expanded indication for treating cataplexy in adults with narcolepsy, making it the only FDA-approved, nonscheduled therapy for the condition.
Cataplexy is a common, debilitating disorder in patients with narcolepsy, marked by sudden and temporary loss of muscle tone that can manifest as mild symptoms (e.g., drooping eyelids) or severely (e.g., body collapse). Cataplexy and excessive daytime sleepiness are defining conditions for the neurologic disorder narcolepsy, in which sleep-wake stability is disrupted.
Wakix received FDA approval in August 2019 as a therapy for excessive daytime sleepiness in adults with narcolepsy. It had received orphan drug designation for the treatment of narcolepsy in 2010.
The drug is a selective histamine 3 (H₃) receptor antagonist/inverse agonist. It increases the production and release of histamine, which promotes wakefulness.
Venclexta combination approved for untreated AML
AbbVie and Genentech’s Venclexta (venetoclax tablets) has been approved in combination with azacitidine, decitabine, or low-dose cytarabine for newly diagnosed acute myeloid leukemia (AML) in adults aged 75 years or older or patients with comorbidities that preclude treatment with intensive induction chemotherapy.
Venclexta was originally approved in 2016 for chronic lymphocytic leukemia (CML) with 17p deletion. Since then, it has also been approved for previously treated CML, newly diagnosed AML, and chronic lymphocytic leukemia. The therapy was initially granted accelerated approval for this indication in November 2018.
The review for this latest approval was conducted under Project Orbis, together with the Australian Therapeutic Goods Administration, the Brazilian Health Regulatory Agency, Health Canada, and Swissmedic. The reviews are still underway in the other agencies.
In addition, the review used the real-time oncology review pilot program and the assessment aid. The agency granted the application priority review, as well as breakthrough and orphan drug designations.
Keytruda nabs new indication for classical Hodgkin lymphoma
Merck Sharp’s Keytruda (pembrolizumab injection) has been granted extended approval for adults with relapsed or refractory classical Hodgkin lymphoma (cHL) and children with refractory cHL or cHL that has relapsed after two or more lines of therapy.
Keytruda was first approved in 2014 for unresectable or metastatic melanoma with disease progression despite previous treatment. It has since been approved for numerous diseases, including lung cancer, gastric cancer, and primary mediastinal large B-cell lymphoma.
In this latest action, the FDA collaborated with the Australian Therapeutics Goods Administration and Health Canada in the review process, as part of Project Orbis. The review is still underway for the Australian and Canadian agencies. This review also used the assessment aid.
The application was granted orphan drug and breakthrough therapy designations, as well as priority review.