Tag Archive for: rare disease

The California-based biotech also announced that it has filed a supplemental New Drug Application (sNDA) seeking approval for a higher concentration formulation of Livmarli for younger patients with progressive familial intrahepatic cholestasis (PFIC), a rare genetic disorder that causes progressive liver disease and can lead to significant morbidity and mortality. Mirum hopes to introduce this higher-concentration regimen later this year, according to its announcement.

Revenue from AstraZeneca’s rare diseases portfolio, boosted by the $39 billion acquisition of Alexion in 2021, has swelled in recent years, to nearly $7.8 billion in 2023.

The company stated that it acquired the exclusive marketing rights for Europe for a new drug that is under regulatory review to treat a rare, potentially fatal heart disease for up to $310 million upfront.

Rare diseases currently afflict 300 million people worldwide and 30 million people in the U.S. alone. However, 95% of these diseases lacked an FDA-approved treatment as of January 2023. One reason, industry leaders say, is cost.

The U.S. Department of Justice has slapped BioMarin Pharmaceutical with a subpoena in connection with its sponsored testing programs for the rare disease therapies Vivizim and Naglazyme.

The deal will bolster nthye French company’s drug development portfolio with an experimental treatment for a rare genetic disease.

Approaching the start of their 7th year in business as a full-service healthcare agency, QBFox Healthcomm is taking on the new year with renewed resolve. Driven by new business, a key promotion, and expanding staff, the agency continues on its roll.

A legislative proposal, if passed into law, could cost the United States up to 1.2 million jobs and lead to more than 130 fewer FDA approvals over a 10-year period.

Iovance’s lifileucel for advanced melanoma and Madrigal’s resmetirom for nonalcoholic steatohepatitis are just a couple of the key decisions on the FDA’s docket this quarter.

The drug, branded as Wainua, is approved for patients with polyneuropathy, or nerve damage caused by hereditary transthyretin amyloidosis (ATTR-PN), which affects an estimated 40,000 patients globally.