The 71st annual American College of Cardiology’s Scientific Session saw several wins – and some “mehs” led by some of biopharma’s largest companies, according to BioSpace.

The U.S. health regulator approved Marinus Pharmaceuticals Inc.’s lead drug to treat seizures associated with a rare genetic disorder in patients two years of age and older, the company said on March 18, sending its shares soaring around 36 percent.

AstraZeneca’s rare disease arm Alexion agreed to resolve all patent disputes with Chugai Pharmaceutical regarding the prescription drug Ultomiris (ravulizumab) by signing a settlement agreement that will involve a one-time payment of $775 million. 

On International Women’s Day, the Valuable 500 – the largest network of global CEOs committed to disability inclusion – announced the launch of the Valuable Directory, convening business disability experts from across the globe. Rare Patient Voice LLC, a leading firm connecting people with disabilities to opportunities for sharing their voices with researchers, is listed in this unique resource featuring expertise within digital, physical and cognitive accessibility, as well as tools for inclusive recruitment and branding practices. 

In recognition of International Women’s Day 2022 on March 8, members of the Rare Patient Voice leadership team reflected on female leadership, team work, role models and lessons they have learned along the way.

Takeda

Takeda and Code Biotherapeutics announced a collaboration agreement to leverage the latter’s proprietary targeted 3DNA non-viral genetic medicine delivery platform for the design and development of gene therapies for rare diseases.

Drew Desjardins, Avalere Health

Historically, therapies focusing on niche markets face tremendous obstacles, from drug development and clinical trials to drug access. Drew Desjardins – EVP, Chief Strategy Officer, Fishawack Health – analyzes several key challenges for brand marketers readying to launch products in these small markets, along with solutions for driving better stakeholder experiences.  

Rare Patient Voice LLC, a leading patient recruitment firm, and MediFind, an advanced digital health platform helping people facing complex health challenges find better care, faster, published a report examining the current state of the diagnostic odyssey for patients with complex diseases during these times of global health issues.

Remember back in March and April and May of 2020, when people prefaced their plans for the future with, “When we get back to normal”? We never did, and we never will. The COVID experience has forever changed how all of us view the world, how we interact with each other … and, of course, how we interact with healthcare brands and what we expect from them. In seeking out insights from pharma’s wise heads for Med Ad News’ annual Agenda report, we tried to maneuver around COVID at least a little, to see what else was going on in the industry. But the plain fact is that we couldn’t, not really.

Shares of Taysha Gene Therapies were down more than 10 percent after a patient’s death marred positive interim data for the company’s experimental gene therapy treatment for Sandhoff and Tay-Sachs diseases, which are two forms of GM2 gangliosidosis.