ClinicaSpace

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several companies—including Novartis, Scholar Rock and Biogen—progressing novel candidates through clinical trials.

The FDA approval of Alnylam’s Amvuttra sets up a three-way race with Pfizer and BridgeBio, which both market transthyretin stabilizers for transthyretin amyloid cardiomyopathy.

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.

Having established success in cancer, biopharma is now looking to leverage CAR T therapies against a new target, autoimmune disorders, with several early- to mid-stage readouts expected this year.

Ionis and Ultragenyx are competing to develop oligonucleotide treatments for Angelman syndrome, but will Neuren’s peptide catch up?

As obesity drug developers compete for the highest weight-loss efficacy, experts contend that overall health outcomes—evidenced by successful studies in therapeutic areas like cardiovascular and sleep apnea—may prove a greater market advantage.

In this deep dive, BioSpace explores the diverse therapeutic modalities now in development, as well as the opportunities and battles for market dominance in this emerging space.

While drug developers work to mitigate the side effects associated with GLP-1–based obesity drugs, recent studies reveal that myriad variables are causing patients to stop treatment.

As sales of its COVID vaccine plummet, Novavax is looking ahead toward other novel vaccines, brought to market with the help of the company’s pharma partners—something it opted not to do as the pandemic swept the globe in 2020.

The FDA’s Oncologic Drugs Advisory Committee recently voted to narrow the label for checkpoint inhibitors Keytruda and Opdivo in stomach and esophageal cancers based on PD-L1 expression levels—but the high unmet need in these patient populations should also be considered.