With three rare pediatric disease priority review vouchers (RPD PRVs) awarded just since the end of March, the nearly year-and-a-half lapse in the program’s reauthorization seems to have had little short-term impact. The three new vouchers bring the total RPD PRVs granted so far this year to seven – one more than the agency issued all last year and down two from the nine given in 2024.
With the U.S. FDA’s approval of GSK plc’s ileal bile acid transporter (IBAT) inhibitor, Lynavoy (linerixibat), patients with primary biliary cholangitis no longer need off-label treatments for a debilitating internal itch symptom called cholestatic pruritus.
Otsuka Pharmaceutical Co. Ltd. reaped positive results from an open-label phase II study of repinatrabit (JNT-517) in adolescents with phenylketonuria (PKU), a rare disease marked by the inability to break down the amino acid phenylalanine.
Otsuka Pharmaceutical Co. Ltd. reaped positive results from an open-label phase II study of repinatrabit (JNT-517) in adolescents with phenylketonuria (PKU), a rare disease marked by the inability to break down the amino acid phenylalanine.
At the current pace of innovation in the U.S. rare disease space, developing and approving therapies for just half of the 10,000-plus known rare diseases would take more than 160 years, Bradley Campbell, president and CEO of Amicus Therapeutics Inc., recently told the Senate Committee on Aging.
A lot of distance lies between talking regulatory flexibility and actually being flexible. That message was driven home again after Uniqure NV disclosed in its latest earnings report March 2 that the U.S. FDA wants a sham-controlled study before it will consider approval of the company’s gene therapy AMT-130 in Huntington’s, a rare disease currently affecting about 41,000 people in the U.S.
Tessera Therapeutics Inc.’s lead in vivo gene editing program, TSRA-196, has been awarded orphan drug and fast track designations by the FDA for adults with α-1 antitrypsin deficiency (AATD).
The pressure on U.S. drug prices continues, with the CMS lining up the drugs for round 3 of negotiations, which will set maximum fair prices to go into effect in 2028. The slate includes 15 drugs and, for the first time, opens the negotiations to Part B drugs, as well as Part D. Consequently, seven of the 15 selected drugs are biologics.
Adolore Biotherapeutics Inc. has announced that the FDA has granted orphan drug designation to the company’s Kv7-activating rdHSV-CA8* gene therapy for treatment of primary and secondary erythromelalgia.
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