Drug Pipeline

Two drugs that target the c-MET protein are in phase 3 trials.

Although Aduhelm stumbled, Leqembi has received full FDA approval, and Eli Lilly has reported positive results for donanemab.

Selective estrogen receptor degraders are vying to join aromatase inhibitors and selective estrogen receptor modulators as therapy after the initial treatment of breast cancer.

The approval of Mounjaro (tirzepatide) for weight loss could be a tipping point for weight-loss drugs.

Ocaliva and resmetirom may be approved by the FDA this year.

Sarepta Therapeutics and Roche are competing with Pfizer to develop the first gene replacement therapy for Duchenne muscular dystrophy. Sarepta and Roche are ahead after the FDA put a clinical hold on Pfizer’s product.

Three ADCs are in late-stage trials as treatments for patients with non-small cell lung cancer.

Research stalled for decades after deaths in a 1960s trial. Now Pfizer and other companies are reporting results for a vaccine against respiratory syncytial virus infections.

CSL Behring's gene therapy, called EntranaDez, may be approved later this month. BioMarin, Pfizer, Bayer and Freeline Therapeutics also have gene therapies for hemophilia in development.

Gilead’s magrolimab is the leading CD47 immune checkpoint inhibitor in development.

Several drugs with novel mechanisms of action have been put on the pathway for speedier approval decisions by the FDA. Phase 2 and 3 trials are underway.

Three gene therapies in late-stage development are designed to supply working copies of genes that people with hemophilia are missing. They may be one-time, curative treatments, but the seven-figure price tags will be an issue.

Nonsteroidal topical agents and TYK2 inhibitors may join an already full armamentarium.

An OptumRx drug pipeline report highlights Zynteglo, a curative gene therapy treatment for beta-thalassemia, and Skysona, a gene therapy for a cerebral adrenoleukodystrophy. FDA approval decision are imminent for both. Price tags of $2 million are expected.

The review, partly funded by Amgen, the maker of Riabni, makes the case for using the biosimilar to Rituxan for all indications of the originator.

Cuban, an investor on “Shark Tank” and owner of the NBA’s Dallas Mavericks, has launched a namesake online pharmacy that says it will have transparent pricing and will bypass pharmacy benefit managers.

The race is on to be the first-approved Bruton tyrosine kinase inhibitor. Meanwhile, trials are underway to test statins and metformin as treatments for multiple sclerosis.

Teplizumab is being evaluated to delay type 1 diabetes. The PDUFA date is August 17, 2022.

Getting boosted is now the rule, not the exception, when it comes to COVID-19 vaccination recommendations. The CDC now recommends that everyone, ages 12 and older, get a dose of an mRNA COVID-19 vaccine, either Pfizer’s or Moderna’s. For people who have gotten the two-shot Pfizer or Moderna series, the booster is their third jab. For those who got the single-dose Johnson & Johnson vaccine, it is the second.

Despite the promise of savings billions of dollars in the United States, adoption of biosimilars has been slow. A roundtable discussion among employers highlighted some of the barriers, including formulary design and drug pricing and rebates.

Drug manufacturers are developing long-acting, injectable HIV drugs for both treatment and prevention.

2021 was a year in which investigators, government officials and drug companies overcame immense challenges to develop successful vaccines against COVID-19. That spirit of innovation is expected to carry over into this year.

Prescription digital therapeutics are FDA approved and available only by prescription. Their developers are aiming to get them covered by insurers.

Drugs in development and newly approved drugs for non-small cell lung cancer are based on research that has identified driver mutations leading to runaway cell growth and division that characterize cancer.

Safety concerns may limit JAK inhibitors as treatment for the common skin condition.

The FDA rejected Humanigen’s application for an emergency use authorization, but the race is on to develop an effective therapy for the cytokine storms that affect patients with COVID-19.

Data is being presented at the 10th Annual IDWeek Virtual Conference, September 29 to October 3.

The flu program is the first in programs leveraging BioNTech’s mRNA technology for vaccines for other respiratory viruses, oncology, and genetic diseases.

Regeneron also announced an expanded contract with the U.S. government for the purchase of additional doses of REGEN-COV.

Point32Health is the name of the not-for-profit health care organization formed by the merger of Harvard Pilgrim Health Care and Tufts Health Plan.