Anticipated cost savings, including a net workforce reduction of approximately 27% in 2025, support company operations into 1H 2027 and through anticipated, first commercial launch in the U.S.
All entries for: Rare Disease
January 9, 2025
Intellia Therapeutics
Layoffs
Cambridge, MA
501-1,000 employees
Disease Area: Rare Diseases
Drug Type: Biologic, Small Molecule
January 8, 2025
Galapagos
Layoffs
Cambridge, MA
1,001-5,000 employees
Galapagos intends to reorganize its business to focus on long-term value creation in cell therapy in oncology. This is anticipated to lead to a reduction of approximately 300 positions.
Disease Area: Rare Diseases
Drug Type: Biologic, Small Molecule
December 31, 2024
Kiora Pharmaceuticals
Negative Outlook
Encinitas, CA
1-50 employees
“Current laws, as well as other healthcare reform measures that may be adopted in the future, may result in more rigorous coverage criteria and in additional downward pressure on the price that we receive for any approved product.”
Disease Area: Rare Diseases
Drug Type: Biologic, Small Molecule
December 9, 2024
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
Disease Area: Multiple, Oncology, Rare Diseases
Drug Type: Small Molecule
November 14, 2024
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
Disease Area: Rare Diseases
Drug Type: Small Molecule
April 24, 2024
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
Disease Area: Cardiology, Chronic Disease, Multiple, Rare Diseases
Drug Type: Biologic, Small Molecule
November 2, 2023
Mirum Pharmaceuticals
Negative Outlook
Foster City, CA
51-200 employees
“We expect that the Affordable Care Act, the IRA and other healthcare reform measures that may be adopted in the future may result in additional reductions in Medicare and other healthcare funding, more rigorous coverage criteria, new payment methodologies and additional downward pressure on the price that we receive for any approved product. Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from third-party payors. The implementation of cost containment measures or other healthcare reforms may prevent us from being able to generate revenue, attain profitability or commercialize our approved medicines and our product candidates, if approved.”
Disease Area: Rare Diseases
Drug Type: Biologic
August 7, 2023
Mirum Pharmaceuticals
Negative Outlook
Foster City, CA
51-200 employees
“It is currently unclear how the IRA will be effectuated but is likely to have a significant impact on the pharmaceutical industry.”
Disease Area: Rare Diseases
Drug Type: Biologic
May 4, 2023
Mirum Pharmaceuticals
Negative Outlook
Foster City, CA
51-200 employees
“It is currently unclear how the IRA will be effectuated but is likely to have a significant impact on the pharmaceutical industry.”
Disease Area: Rare Diseases
Drug Type: Biologic
November 9, 2022
Mirum Pharmaceuticals
Negative Outlook
Foster City, CA
51-200 employees
Likely to Have “Significant Impact”: “These provisions will take effect progressively starting in 2023, although they may be subject to legal challenges. It is currently unclear how the IRA will be effectuated but is likely to have a significant impact on the pharmaceutical industry” Impact to Livmarli: “Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from third-party payors. The implementation of cost containment measures or other healthcare reforms may prevent us from being able to generate revenue, attain profitability or commercialize Livmarli and our other product candidates, if approved.”
Disease Area: Rare Diseases
Drug Type: Biologic
