FDA Action Alert: Acer & Relief, Sanofi & Regeneron, Pfizer-BioNTech & Moderna and Rhythm

FDA_Sarah Silbiger/Getty Images

Sarah Silbiger/Getty Images

June is starting off with a number of important dates for the U.S. Food and Drug Administration, including an advisory committee meeting to discuss COVID-19 vaccines in children as young as six months. Continue reading for details.

Acer and Relief’s Urea Cycle Disorders Drug 

Acer Therapeutics and Relief Therapeutics have a target action date of June 5 for their ACER-001 (sodium phenylbutyrate) for the treatment of patients with Urea Cycle Disorders (UCDs). The application is supported by data from two bioequivalence trials where the drug demonstrated similar relative bioavailability for both phenylbutyrate (PBA) and phenylacetate (PAA) compared to the reference drug, Buphenyl (sodium phenylbutyrate).

UCDs are a group of illnesses generated by genetic mutations that cause a deficiency in one of the six enzymes that catalyze the urea cycle, leading to excess accumulation of ammonia in the bloodstream. Acute hyperammonemia can result in lethargy, somnolence, coma and multiple organ failure. Chronic hyperammonemia can cause headaches, confusion, lethargy, failure to thrive, behavioral changes and learning and cognitive problems.

Sanofi and Regeneron’s Dupixent for Atopic Dermatitis in Little Kids

Sanofi and Regeneron have a target action date of June 9. This is for their supplemental Biologics License Application (sBLA) for Dupixent (dupilumab) as an add-on maintenance treatment for children aged six months to five years with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription drugs or when those therapies aren’t recommended.

The sBLA is supported by results from the pivotal Phase III study of the drug added to standard-of-care topical corticosteroids in this age group. The study hit all primary and secondary endpoints, demonstrating that Dupixent and the steroids significantly improved skin clearance and decreased overall disease severity and itch at 16 weeks compared to steroids alone. The sBLA is under Priority Review.

FDA Vaccines Adcomm to Discuss Moderna and Pfizer-BioNTech COVID-19 Vaccines for Kids

The FDA’s Vaccines and Related Biological Products Advisory Committee is meeting on June 14 to discuss amending Moderna’s Emergency Use Authorization (EUA) for its COVID-19 vaccine to include the primary two-shot series in children and adolescents six years through 17 years. Then on June 15, it will meet to discuss the primary series in infants and children six months through five years of age. The committee will also discuss amending Pfizer and BioNTech’s EUA to include the primary series of their mRNA COVID-19 vaccine for infants and children six months through four years of age.

In their studies, Pfizer and BioNTech indicated that their three-dose vaccine for kids ages six months through four was 80% effective at preventing the disease from the Omicron variant. They are three micrograms, one-tenth the dose given to adults. Moderna indicated its two-dose vaccine is about 51% effective against infection from Omicron in children under two and about 37% effective in the two through five age group. The company believes the vaccine should provide high levels of protection against severe disease. Moderna’s doses for kids are 25 micrograms, a quarter of the 100 micrograms approved for adults.

Rhythm’s Imcivree for Bardet-Biedl and Alström Syndromes

Rhythm Pharmaceuticals has a target action date of June 16 regarding its sNDA for Imcivree (setmelanotide) for the treatment of obesity and hunger control in adult and pediatric patients six years and older with Bardet-Biedl Syndrome (BBS) or Alström syndrome. On February 23, the agency informed Rhythm that its original PDUFA date had been pushed back to June 16 after requesting additional subgroup analyses of the clinical efficacy data from its Phase III trial in BBS and Alström syndrome. No new data were requested.

In the U.S., Imcivree is approved for chronic weight management in adults and children six years and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisn/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency.

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