Why it matters: Orphan drugs are exempt from price controls in the Inflation Reduction Act—only if they’re approved for a single indication, disincentivizing development. The bill would protect drugs for orphan and rare diseases from price controls even if they’re used for multiple indications.
BIO’s view: “BIO looks forward to working with lawmakers to get the (ORPHAN Cures Act) across the finish line as quickly as possible,” BIO CEO Rachel King previously said. “Reversing IRA’s perverse incentives will eliminate the significant barrier for scientists to usher in new waves of rare disease drug innovation.”
A case-in-point on orphan cures: FDA approves Servier drug for rare cancer
TIBSOVO, already approved for several other indications, received Food and Drug Administration (FDA) approval for use against a rare blood cancer.
Why it matters: TIBSOVO, developed by BIO member Servier, became the only therapy approved for adults with relapsed or refractory myelodysplastic syndromes (MDS) with an isocitrate dehydrogenase-1 (IDH1) mutation.
Other indications: TIBSOVO already has FDA approval for use against other conditions existing in combination with the IDH1 mutation: newly diagnosed AML, relapsed or refractory AML, and locally advanced or metastatic cholangiocarcinoma.
Why other indications matter: The Inflation Reduction Act currently eliminates incentives for treating rare diseases by investigating new uses for existing drugs. The ORPHAN Cures Act introduced in the Senate yesterday and the House last month would reinstate these incentives.
What they’re saying: “Servier is proud to lead the way in mutant IDH inhibition through continued innovations that support patients living with difficult and hard-to-treat cancers,” said Servier’s Arjun Prasad. “FDA approval for TIBSOVO reinforces our commitment to deliver significant advances in areas of high unmet need.”
BIO Beltway Report
President Biden’s Friday: A campaign reception in Washington before heading to Delaware for the weekend.
What’s Happening on Capitol Hill: Enjoy the weekend.
Biotechnology Innovation Organization (BIO)
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