News Updates
Tracking Rare Disease Drug Development Challenges Amid a Shifting Policy Environment
Rare diseases are defined as those which affect fewer than 200,000 people in the United States. Despite advancements in rare disease research and development due to incentives created in the Orphan Drug Act more than 40 years ago, many rare diseases still lack effective treatments due to the complex science and limited market incentives involved in their development. Of the 10,000+ known rare diseases, less than 10% have an FDA-approved treatment. The Inflation Reduction Act's healthcare provisions are an example of policies that discourage investment in rare disease treatments, leading to discontinued research, layoffs, and ultimately delayed treatments for patients. Incubate's Rare Disease Investment Tracker measures the impacts of a challenging policy environment on rare disease R&D.
Daiichi Sankyo
Discontinued Research
Basking Ridge, NJ
10,001-50,000 employees
A study on the efficacy of DS-2325a for patients with Netherton Syndrome was terminated based on a business decision by the sponsor.
1 Discontinued Research Program
Epizyme
Discontinued Research
Boston, MA
51-200 employees
Epizyme, Inc. has terminated its study of tazemetostat with enzalutamide or abiraterone/prednisone in participants with advanced prostate cancer (CELLO-1) due to a sponsor decision unrelated to safety concerns.
1 Discontinued Research Program
ApcinteX
Discontinued Research
United Kingdom
1-50 employees
A study on SerpinPC for patients with severe hemophilia A or moderately severe to severe hemophilia B was terminated due to a business and strategic decision.
1 Discontinued Research Program
BioMarin
Discontinued Drug, Discontinued Research, Layoffs
San Rafael, CA
1,001-5,000 employees
As a result of its prioritized portfolio, four programs will be discontinued, including BMN 331, BMN 255, BMN 355 and BMN 365. None of the programs were discontinued due to safety signals.
4 Discontinued Drugs: BMN 331 (biologic, orphan), BMN 255 (small molecule), BMN 355 (biologic), BMN 365 (biologic)
4 Discontinued Research Programs
Alnylam
Discontinued Drug, Discontinued Research
Cambridge, MA
1,001-5,000 employees
“In addition to the updated Helios-B plan, Alnylam also announced Thursday that it’s having second thoughts about developing RNA interference therapy for Stargardt disease, a rare genetic eye disease that can cause vision loss. Alnylam said it will not launch a phase 3 study of Amvuttra in Stargardt in late 2022 as previously announced.
“Alnylam attributed the pause to Biden’s Inflation Reduction Act, which allows Medicare to directly negotiate prices of some high-expenditure drugs. Drugs with one single orphan drug designation is exempt from potential price negotiations, a term that may discourage companies from exploring approvals in additional indications, Alnylam CEO Yvonne Greenstreet noted on the call. Because Amvuttra and Onapttro already has an orphan status in ATTR, an additional orphan label could theoretically open it for potential pricing scrutiny.”
1 Discontinued Drug: Amvuttra (biologic) in Stargardt disease
1 Discontinued Research Program
