Approval Of Drug For Friedreich’s Ataxia Could Open Door For Other Researchers Of Rare Illnesses
Read about the biggest pharmaceutical developments and pricing stories from the past week in KHN's Prescription Drug Watch roundup.
Stat:
FDA Approves Reata's Treatment For Rare Neurological Disease
The Food and Drug Administration on Tuesday approved the first drug to treat Friedreich’s ataxia, a rare, neurological disease. The new drug, called Skyclarys, is made by Reata Pharmaceuticals. (Feuerstein, 2/28)
The Wall Street Journal:
FDA Widens Path For Rare-Disease Treatments With New Approval
The FDA typically requires results from two clinical trials demonstrating a drug’s efficacy to ensure positive results in one trial are replicable. But with rare conditions, finding enough patients to run two clinical trials can be daunting, said Jennifer Farmer, chief executive officer of the Friedreich’s Ataxia Research Alliance, which funds research on the disease, including the natural history study. Families of patients have raised about $1 million a year to fund the study, Ms. Farmer said. (Marcus, 2/28)
In other pharmaceutical developments —
Reuters:
Jazz Pharma Ruling Clears U.S. Roadblock For Rival Narcolepsy Drug
A U.S. appeals court ordered Jazz Pharmaceuticals Inc on Friday to de-list a patent related to its blockbuster narcolepsy drug Xyrem from the U.S. Food and Drug Administration's register of approved medications, in a win for rival drugmaker Avadel CNS Pharmaceuticals Inc. (Brittain, 2/26)
FiercePharma:
Cytokinetics’ Heart Failure Drug Gets A Thumbs Down From FDA
With an innovative approach to treating heart failure, Cytokinetics touted a game-changing medicine that could someday become available for an elusive condition that effects more than 6 million in the U.S. (Dunleavy, 2/28)
FiercePharma:
Novartis Halts Pluvicto New Patient Starts Amid Supply Struggle
For patients with metastatic castration-resistant prostate cancer (mCRPC) who have failed on prior treatments, Novartis’ radiotherapy Pluvicto offers a novel option. But the drug can be hard to come by these days, with supply constraints dogging existing patients and also delaying treatment for potential new takers. (Liu, 2/28)
ScienceDaily:
New Purification Method Could Make Protein Drugs Cheaper
Engineers devised a way to purify protein drugs during manufacturing. Their approach, which uses nanoparticles to rapidly crystallize proteins, could help make protein drugs more affordable and accessible, especially in developing countries. (Massachusetts Institute of Technology, 2/28)
