Less than a year after submitting their NDA and several weeks before the June 28 PFUFA date, Bausch + Lomb Corp. and partner Novaliq GmbH have been awarded U.S. FDA approval for Miebo, their perfluorohexyloctane eye drop formulation designed to treat the signs and symptoms of dry eye disease (DED) associated with meibomian gland dysfunction (MGD). Miebo – formerly known as NOV-03 – is the first eye drop on the market to directly target tear evaporation, according to the companies. DED is one of the most common ocular surface disorders, and the discomfort and visual symptoms it causes can impair patients’ ability to perform everyday tasks. Characterized by a deficient tear film lipid layer that leads to increased tear evaporation, MGD is a leading trigger of dry eye. 

Adcom mulls Intercept’s approved OCA given at higher dose for NASH

Shares of Intercept Pharmaceuticals Inc. (NASDAQ:ICPT) halted trading while the U.S. FDA’s Gastrointestinal Drugs Advisory Committee deliberated over obeticholic acid (OCA) at the 25-mg dose for nonalcoholic steatohepatitis. Ocaliva, the 5- and 10-mg formulation of OCA, gained accelerated approval with a boxed warning in 2016 for primary biliary cholangitis in adults. Briefing documents ahead of the meeting suggested a bumpy ride for OCA due to potential liver toxicity and other concerns.

Rona and Keymed partner to develop siRNA therapies for severe kidney diseases

Rona Therapeutics Co. Ltd. and Keymed Biosciences Co. Ltd. formed a collaboration to jointly discover and develop first-in-class siRNA therapeutics for glomerulonephritis, also known as severe kidney diseases. The partnership forged between Rona, of Shanghai, and Keymed, of Chengdu, China, will leverage both companies’ scientific expertise and will build on Rona’s RNA platform targeting liver and extrahepatic tissues. Terms call for Rona to use its Razor platform to discover and identify siRNA lead compounds directed to a novel target related to glomerulonephritis. Keymed will contribute research and translational expertise and capabilities.

Fewer biopharma deals raise similar value year-over-year, while M&A value declines

Biopharma has experienced relatively stable deal value year-over-year, despite a 24% decline in the number of deals through April. M&As, however, have brought in the lowest amount in years. Deal values are down by less than 1% compared to last year: 456 deals have brought in a potential $62.08 billion through the end of April 2023, compared to 600 deals worth $62.46 billion by the same point in 2022. Completed M&As through April are worth $13.36 billion, down 35.37% from 2022’s $20.67 billion.

Adcom backs Pfizer’s RSV vaccine for maternal immunization

Efficacy data for Pfizer Inc.’s respiratory syncytial virus (RSV) vaccine proved convincing for members of the U.S. FDA’s Vaccines and Related Biological Products Advisory Committee, which voted 14-0 May 18 in favor of Abrysvo’s effectiveness when administered during the second or third trimester of pregnancy to protect infants from birth to 6 months, with the adcom endorsing safety data in a 10-4 vote. An FDA decision is due on the priority review BLA in August 2023. Should it follow the recommendations of its adcom, Abrysvo would be the first RSV vaccine available for maternal administration to protect newborns.

Gene editing advances progress, by moving three steps forward and two steps back

The discovery of DNA was a milestone in the history of science that led to a breakthrough in biomedical research. By associating disease and genetics, genome correction techniques were ultimately developed that are supposed to work in the same way that antibiotics and antivirals block pathogenic microorganisms: by directly attacking the causes of disease. However, these scientific contributions pose other challenges and scientists often take steps forward and backward to solve them. Gene editing is precise and selective, but it is not perfect. The enzymes of the CRISPR-Cas9 system break DNA chains or cause DNA modifications in undesired places, off-target editions that call into question the safety of the technique if it is intended to be used in clinical applications.

Also in the news

Alligator, Ambrx, Antengene, Aslan, Avrobio, Biontech, Carsgen, Cellectis, Cognition, Curevac, Eli Lilly, Fibrogen, Fusion Antibodies, Galera, Immupharma, Intensity, Kymera, Kynos, Mindmed, Mirum, Nuformix, Oncosec, Orapharma, Okyo, PDS, Pepgen, Pfizer, Sareum, Vifor, Westvac