Harmony Biosciences Gets FDA Orphan Designation for Pitolisant in Prader-Willi Syndrome
By Colin Kellaher
Harmony Biosciences Holdings has won orphan-drug designation from the U.S. Food and Drug Administration for its pitolisant drug candidate for the treatment of the genetic disorder Prader-Willi syndrome.
The Plymouth Meeting, Pa., pharmaceutical company said it plans to assess the safety and efficacy of pitolisant in treating excessive daytime sleepiness and behavioral disturbances in patients with Prader-Willi in a Phase 3 registrational study slated to launch during the first quarter.
The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S. and provides for an extended marketing exclusivity period against competition.
Harmony said 15,000 to 20,000 people in the U.S. are living with Prader-Willi, a rare neurological disorder with symptoms that result from dysfunction of the part of the brain that controls sleep-wake state stability and signals that mediate the balance between hunger and satiety.
Write to Colin Kellaher at colin.kellaher@wsj.com
(END) Dow Jones Newswires
February 20, 2024 08:34 ET (13:34 GMT)
Copyright (c) 2024 Dow Jones & Company, Inc.-
What’s the Difference Between the CPI and PCE Indexes?
-
Micron Earnings: Great Guidance but Stock Now Looks Fairly Valued
-
August PCE Report Forecasts Show More Good News on Inflation
-
AI Stocks May Be Down, but Don’t Count Them Out
-
4 Stocks to Buy as the Fed Cuts Interest Rates
-
Markets Brief: The Uncertain Path to Neutral Interest Rates
-
What’s Happening in the Markets This Week
-
Where Top Stock Fund Managers Are Looking Next After the Fed Rate Cut
-
Our Top Pick for Investing in US Renewable Energy
-
Undervalued by 25% and Yielding 5%, This Stock Is a Buy
-
Can AI Predict Future Stock Returns?
-
The Best Energy Stocks to Buy Now
-
10 Undervalued Wide-Moat Stocks
-
Obesity Drugs: Can New Firms Take Market Share From Eli Lilly and Novo Nordisk?
-
New 4-Star Stocks
-
Intel Fair Value Left Unchanged Despite Qualcomm Takeover Talk