Cullinan Therapeutics Receives FDA Orphan Drug Designation for CLN-049, a Novel FLT3xCD3 T Cell Engager, in Relapsed/Refractory Acute Myeloid Leukemia

Orphan Drug Designation underscores the potential of CLN-049, a novel FLT3xCD3 T cell engager, to address significant unmet need in AMLCAMBRIDGE, ...

Belite Bio Receives Orphan Drug Status for Tinlarebant in Stargardt Disease in Switzerland

Tinlarebant is the first therapeutic candidate to demonstrate clinical efficacy in Stargardt disease, having met the primary endpoint, reductions ...

Lundbeck receives orphan drug designation in Japan for asedebart for the treatment of patients with congenital adrenal hyperplasia and Cushing's disease

Lundbeck today announced that orphan drug designation (ODD) has been granted in Japan by the Ministry of Health, Labour and Welfare (MHLW) for asedebart (Lu AG13909) for the treatment of patients with ...
JD Supra

Congress Expands Orphan Drug Exemptions From Medicare Price Negotiations

The One Big Beautiful Bill Act ("OBBB") expands exemptions for orphan drugs from mandatory Medicare price negotiations and modifies their eligibility timeline. On July 4, 2025, President Trump signed ...
Wall Street Journal

Orphan Drugs Are Neglected No More

Journal Editorial Report: RFK Jr's new picks play into his critics’ hands. A bill as large as the GOP reconciliation budget takes time to pore through, and we can now report a hidden gem: It fixes the ...
Healthcare Finance News

Orphan drugs exempted from price negotiations to cost Medicare $8.8 billion

The One Big Beautiful Bill Act removed a number of orphan drugs from being subject to price negotiations, and this could cost the Medicare program $8.8 billion over the course of about 10 years. That ...
JD Supra

Potential Effects of the Inflation Reduction Act on Orphan Drug Development

Jonathan Caplan, Jeffrey Coleman Ph.D. President Biden signed the Inflation Reduction Act (IRA) into law on Aug. 16, 2022, with the goal of reducing health care costs for the government and ...
STAT

Pharma companies and their allies seek to exempt orphan drugs from state pricing limits

As more states create dedicated boards to cap the costs of medicines, some drugmakers and their allies are pushing back with a controversial tactic — lobbying for legislation to set exemptions for ...

NV-387 for The Treatment of Measles is Granted Orphan Drug Designation by The US FDA

SHELTON, CT / ACCESS Newswire / May 4, 2026 / NanoViricides, Inc., a publicly traded company (NYSE American:NNVC) (the ...
The American Journal of Managed Care

Payer Coverage of Orphan Drugs Often Varies, According to Research Published in The American Journal of Managed Care®

Rising number, cost of drugs for rare disease therapies create new pressure on health plans, researchers report in the October issue of The American Journal of Managed Care®. (CRANBURY, N.J.) — Drugs ...
The Spokesman-Review

‘It’s just bad luck’: Rising pharmaceutical costs, especially for ‘orphan drugs,’ driving 36% increase in Spokane’s health premiums

Spokane city employees face a 36% increase in health insurance premiums in the coming years as City Hall grapples with soaring pharmaceutical costs, particularly for a category of prescriptions called ...
Health Affairs

Congress Should Remove The Rare Disease Carve-Out From Medicare Drug Price Negotiation, Not Expand It

The Inflation Reduction Act (IRA) of 2022 allowed the Centers for Medicare and Medicaid Services to begin negotiating prices for certain prescription drugs in Medicare. For products to be eligible, ...