FDA Action Alert: Merck, Ligand and Albireo Pharma


There are two PDUFA dates on the U.S. Food and Drug Administration (FDA)’s calendar for this week. Here’s a look.

Ligand Pharma and Merck’s Pneumococcal Vaccine

Merck and Ligand Pharmaceuticals had a target action date of July 18, 2021, for their 15-variant pneumococcal conjugate vaccine to prevent invasive pneumococcal disease in adults 18 years of age and older. The vaccine, dubbed V114, consists of pneumococcal polysaccharides from 15 serotypes conjugated to a CRM197 carrier protein and includes serotypes 22F and 33F. The FDA approved it under the brand name Vaxneuvance on July 16.

In a January 12, 2021 statement, after the FDA accepted the Biologics License Application (BLA) for V144 under priority review, Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories, stated, “Invasive pneumococcal disease in adults is on the rise in many countries, driven by highly-invasive serotypes including serotype 3, which is included in the currently licensed pneumococcal conjugate vaccine, as well as serotypes not included, such as serotypes 22F and 33F. Building on our nearly 40 years of experience with PNEUMOVAX 23, Pneumococcal Vaccine Polyvalent, we have developed V114 as another potentially important option to help protect more adults from invasive pneumococcal disease, especially those who are at increased risk.”

The BLA was built on data from Phase II and III trials in various adult populations, including healthy adults and people at increased risk, such as those with chronic medical conditions, HIV, and people 65 years of age and older.

On May 20, Merck announced that the vaccine had hit the primary immunogenicity and safety endpoints in two Phase III trials in children. Both support the potential use of V114 in healthy infants who may have already started a pneumococcal vaccination series with the 13-valent pneumococcal conjugate vaccine (PCV13) which is already available, as well as in a catch-up setting for healthy children who hadn’t received a pneumococcal vaccine or had already received a full or partial one with lower valency pediatric pneumococcal conjugate vaccines.

“Pneumococcal disease continues to cause serious illness and death worldwide in children under the age of 5, despite the positive impact of pneumococcal conjugate vaccination on pediatric case numbers,” Baynes said. “At Merck, our goal is to expand coverage to new serotypes not targeted by currently available pediatric pneumococcal conjugate vaccines, while maintaining a strong immune response to current vaccine serotypes so as to help sustain progress achieved to date.”

Albireo’s Odevixibat for Pruritus in Progressive Familial Intrahepatic Cholestasis

Alberio Pharma has a target action date of July 20 for its New Drug Application (NDA) for odevixibat pruritis patients with progressive familial intrahepatic cholestasis (PFIC). Pruritis is itchy skin. PFIC results in progressive liver disease that usually leads to liver failure. In PFIC, liver cells have limited ability to secrete bile, which is a digestive fluid. The accumulation of bile in liver cells causes liver disease.

The NDA was built on data from PEDFIC 1 and 2 Phase III studies. In PEDFIC 1, the drug met both the pruritus and serum bile acid primary endpoints. It was also well tolerated with a very low incidence of diarrhea/frequent bowel movements compared to placebo. PEDFIC 2 is an open-label extension study.

Odevixibat is a potent, once-daily, non-systemic IBATi, and acts locally in the small intestines.

On June 21, the company announced it was presenting new data in nine abstracts at the EASL International Liver Congress 2021 on June 23-26. All of the data demonstrated continued support of odevixibat (Bylavy)’s efficacy and tolerability. The data supported a sustained and durable effect, while also demonstrating sleep improvements, as well as growth and hepatic parameters in patients with PFIC.

“We are pleased to follow-up the statistically significant PEDFIC Phase III results with additional detailed data that reinforces the rapid, as well as longer term benefits of Bylvay for patients with PFIC,” said Ron Cooper, president and chief executive officer of Albireo. “The PEDFIC program is the largest body of prospective data collected in patients with PFIC and provides further proof of Albireo’s scientific expertise in applying bile acid modulation to treat rare cholestatic liver disease.”

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