Tag Archive for: rare disease

“What has struck me the most is the pace of change. It is so much faster than when I started, says Italia Marr, director, media strategy at Greater Than One. “Thanks to advanced technology, marketers must keep up more than ever.  It feels like every day, every quarter, every year, there’s new technology that enables novel marketing solutions for our clients.” Marr talks about her latest role at GTO and shares her perspective on healthcare marketing strategy and working in the industry today.

Yesterday Applied Therapeutics announced that the FDA is extending the review period for its investigational aldose reductase inhibitor govorestat for the treatment of classic galactosemia. The biotech now expects a verdict by Nov. 28, 2024.

Orchard Therapeutics, a unit of Japan’s Kyowa Kirin , priced its newly approved gene therapy for a rare, life-threatening genetic disease on Wednesday at $4.25 million, making it the most expensive drug in the United States.

Degenhart will be the agency lead on multiple brands, driving the company’s strategic vision and integrating with other AbelsonTaylor Group teams to deliver measurable business success for clients, the core tenet of AT Group’s “Return on Imagination” philosophy. She reports to Lynnette Hunter, executive vice president, director of client services.

Orchard Therapeutics secured the FDA’s first approval for an autologous gene therapy to treat the rare metabolic disease metachromatic leukodystrophy in children.

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.