All entries for: Rare Diseases

April 24, 2024

BioMarin

Discontinued Drug, Discontinued Research, Layoffs

San Rafael, CA
1,001-5,000 employees
1001-5000 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), BMN 255 (biologic), BMN 355 (biologic), BMN 365 (biologic)
4 Discontinued Research Programs

Disease Area: Cardiology, Chronic Disease, Multiple, Rare Diseases
Drug Type: Biologic
November 2, 2023

Mirum Pharmaceuticals

Negative Outlook

Foster City, CA
51-200 employees
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
51-200 employees
Disease Area: Rare Diseases
Drug Type: Biologic
May 4, 2023

Mirum Pharmaceuticals

Negative Outlook

Foster City, CA
51-200 employees
51-200 employees
Disease Area: Rare Diseases
Drug Type: Biologic
November 9, 2022

Mirum Pharmaceuticals

Negative Outlook

Foster City, CA
51-200 employees
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
October 27, 2022

Alnylam

Discontinued Drug, Discontinued Research

Cambridge, MA
1,001-5,000 employees
1001-5000 employees

“Alnylam Pharmaceuticals Inc. said it’s stopping work on a treatment for a rare eye disease because of a new US drug-pricing law with the potential to limit how much it could charge for the medication in the future.

Alnylam will not begin a planned late-stage trial of its drug Amvuttra for Stargardt disease, which causes blindness, while it examines the Inflation Reduction Act, the company said Thursday in its third-quarter earnings statement.”

1 Discontinued Drug: Amvuttra (biologic)
1 Discontinued Research Program

Disease Area: Rare Diseases
Drug Type: Biologic
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