“It is currently unclear how the IRA will be effectuated but is likely to have a significant impact on the pharmaceutical industry.”
All entries for: Rare Disease
August 7, 2023
Mirum Pharmaceuticals
Negative Outlook
Foster City, CA
51-200 employees
Disease Area: Rare Diseases
Drug Type: Biologic
July 28, 2023
Homology Medicines
Discontinued Drug, Discontinued Research, Layoffs
Waltham, MA
1-50 employees
“Homology Medicines will lay off scores of its employees and stop research in a reset meant to position the company for a sale, merger or other business combination.”
1 Discontinued Drug: HMI-203 for treatment of Mucopolysaccharidosis type II (MPS II)
1 Discontinued Research Program
Disease Area: Immune Diseases, 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
October 27, 2022
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
Disease Area: Rare Diseases
Drug Type: Biologic
August 2, 2022
Edgewise Therapeutics
Negative Outlook
Boulder, CO
51-200 employees
“The implementation of cost containment measures, including the prescription drug provisions under the Inflation Reduction Act, as well as other healthcare reforms may prevent us from being able to generate revenue, attain profitability, or commercialize our product candidates.”
Disease Area: Chronic Disease, Rare Diseases
Drug Type: Biologic, Small Molecule
