Chimerix Receives U.S. Food and Drug Administration Approval for TEMBEXA® (brincidofovir) for the Treatment of Smallpox
- Category: Small Molecules
- Published on Sunday, 06 June 2021 12:30
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– First smallpox antiviral approved for all age groups, including infants, and patients who have difficulty swallowing –
DURHAM, NC, USA I June 04, 2021 I Chimerix (NASDAQ:CMRX), a biopharmaceutical company focused on accelerating the development of medicines to treat cancer and other serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted TEMBEXA® (brincidofovir) tablets and oral suspension approval for the treatment of smallpox. TEMBEXA is approved for adult and pediatric patients, including neonates.
“We are delighted to report our first FDA approved products for the treatment of smallpox, particularly as the importance of pandemic preparedness has been put into focus over the last year. With this approval in hand, we now look forward to advancing our discussions with the Biomedical Advanced Research and Development Authority (BARDA) toward a procurement contract to support national preparedness,” said Mike Sherman, Chief Executive Officer of Chimerix.
Chimerix developed the TEMBEXA oral formulations as medical countermeasures for the treatment of smallpox under an ongoing collaboration with BARDA, part of the office of the Assistant Secretary for Preparedness and Response within the U.S. Department of Health and Human Services, under contract number HHSO100201100013C.
TEMBEXA’s approval is based on efficacy data in two lethal orthopoxvirus animal models of human smallpox disease, the rabbitpox model (New Zealand White rabbits infected with rabbitpox virus) and the mousepox model (BALB/c mice infected with ectromelia virus). In the pivotal studies in each model, TEMBEXA treatment resulted in statistically significant survival benefit versus placebo following delayed treatment after animals were infected with a lethal viral dose. The FDA’s ‘Animal Rule’ allows for testing of investigational drugs in animal models to support effectiveness in diseases which are not ethical or feasible to study in humans. The TEMBEXA U.S. Prescribing Information has a BOXED WARNING for increased risk for mortality when used for longer duration; see below for Important Safety Information.
Chimerix is a development-stage biopharmaceutical company dedicated to accelerating the advancement of innovative medicines that make a meaningful impact in the lives of patients living with cancer and other serious diseases. Most recently, the Company obtained FDA approval for brincidofovir as a medical countermeasure for the treatment of smallpox. The Company has two other advanced clinical-stage development programs, ONC201 and dociparstat sodium (DSTAT). ONC201 is currently in a registrational clinical program for recurrent H3 K27M-mutant glioma and a blinded independent central review is expected later in 2021. DSTAT is in development as a potential first-line therapy in acute myeloid leukemia.
Smallpox is a highly contagious disease caused by the variola virus. Historically, smallpox was one of the deadliest diseases in history with a case fatality rate of approximately 30%. Despite successful eradication of smallpox in the 1970s, there is considerable concern that variola virus could reappear, either through accidental release or as a weapon of bioterrorism. According to the U.S. Centers for Disease Control and Prevention (CDC), variola virus is ranked in the highest risk category for bioterrorism agents (Category A) due to its ease of transmission, high mortality rate, and potential to cause public panic and social disruption.
TEMBEXA is an oral antiviral formulated as 100 mg tablets and 10 mg/mL oral suspension dosed once weekly for two weeks. TEMBEXA is indicated for the treatment of human smallpox disease caused by variola virus in adult and pediatric patients, including neonates. TEMBEXA is not indicated for the treatment of diseases other than human smallpox disease. The effectiveness of TEMBEXA for the treatment of smallpox disease has not been determined in humans because adequate and well-controlled field trials have not been feasible and inducing smallpox disease in humans to study the drug’s efficacy is not ethical. TEMBEXA efficacy may be reduced in immunocompromised patients based on studies in immune deficient animals.
TEMBEXA (brincidofovir) is a nucleotide analog lipid-conjugate designed to mimic a natural monoacyl phospholipid to achieve effective intracellular concentrations of the active antiviral metabolite, cidofovir diphosphate. Cidofovir diphosphate exerts its orthopoxvirus antiviral effects by acting as an alternate substrate inhibitor for viral DNA synthesis mediated by viral DNA polymerase.