2023-01-31 12:05:00

Ionis receives FDA Fast Track designation for olezarsen in patients with familial chylomicronemia syndrom

Logo Benzinga
By Pr Newswire
  • FCS is a rareand debilitating genetic disease often leading to significant risk of acute, potentially fatal pancreatitis
  • If approved, olezarsen would be the first approved treatment for FCS in the U.S.

CARLSBAD, Calif., Jan. 31, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. IONS today announced that the U.S. Food and Drug Administration (FDA) has granted olezarsen Fast Track designation for the treatment of familial chylomicronemia syndrome (FCS).

FCS is a debilitating genetic disease characterized by severely high levels of plasma triglyceridesand a risk of unpredictable and potentially fatal acute pancreatitis. In addition to acute pancreatitis, FCS patients are at risk of chronic complications due to permanent organ damage, including chronic pancreatitis and type 3c diabetes. There are currently no approved therapies for the treatment of FCS in the U.S.

Fast Track designation is designed to expedite the FDA's review of innovative, new drugs that demonstrate the potential to address unmet medical need.

Continue read on benzinga.com

Logo PR Newswire
HealthPress Release2023-01-30 15:00:00
BEIJING, Jan. 30, 2023 /PRNewswire/ -- Help Therapeutics Co., Ltd. (hereinafter Help Therapeutics) has revealed that the China Center for Drug Evaluation...

Logo GlobeNewswire
SciencePress Release2023-01-31 11:00:00
~ PEDMARKis the First and Only FDA-Approved Therapy Indicated to Reduce the Risk of Ototoxicity Associated with Cisplatin in Pediatric Patients with...

Logo Nasdaq
Business / FinanceBy Rtt News2023-01-30 12:26:23
(RTTNews) - Xeris Biopharma Holdings Inc. (XERS) said Monday that the U.S. Food and Drug Administration granted its subsidiary Xeris Pharmaceuticals Inc.,...

Logo PR Newswire
HealthPress Release2023-01-30 18:31:00
GERMANTOWN, Md., Jan. 30, 2023 /PRNewswire/ -- Deka Biosciences ("Deka"), a biotech company focused on developing novel cytokine therapies to treat cancer...

Logo GlobeNewswire
SciencePress Release2023-01-30 21:01:00
2022 Success Has Increased Confidence to Focus LNC Delivery Platform on the Future of Medicine - Delivering Genes Prioritization on Building External...

Logo GlobeNewswire
HealthPress Release2023-01-30 13:30:00
Company submitted nearly one quarter of all candidate gene submissions to GeneMatcher in 2022 and collaborated on 63 publications involving new disease-gene...

Logo Benzinga
Business / FinanceBy Business Wire2023-01-31 11:05:00
Arugula Sciences announced today that it has received clearance from the FDA for its investigational new drug (IND) application for a Phase 1, open-label,...

Logo GlobeNewswire
SciencePress Release2023-01-31 12:00:00
Patient screening to initiate; topline data expected by mid-2023 BOCA RATON, Fla., Jan. 31, 2023 (GLOBE NEWSWIRE) -- First Wave BioPharma, Inc. (NASDAQ:FWBI)...

Logo NewsFileCorp
Press Release2023-01-31 12:00:00
San Diego, California--(Newsfile Corp. - January 31, 2023) - Skye Bioscience, Inc. (OTCQB: SKYE) ("Skye" or the "Company"), a pharmaceutical company...

Logo PR Newswire
HealthPress Release2023-01-31 01:00:00
- Leveraging the success in Japan to expand the Alluminox™ platform globally and obtain new approvals as early as 2025 SAN DIEGO, Jan. 30, 2023 /PRNewswire/...

Logo GlobeNewswire
SciencePress Release2023-01-30 13:00:00
Media Release Second US FDA Fast Track designation granted to eftilagimod alpha (efti) supporting planned late-stage development in 1st line non-small cell...

Logo GlobeNewswire
SciencePress Release2023-01-30 23:00:00
LA JOLLA, Calif., Jan. 30, 2023 (GLOBE NEWSWIRE) -- MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the...

Logo GlobeNewswire
SciencePress Release2023-01-30 13:00:00
Commercial launch planned for February 20, 2023 BURLINGTON, Mass., Jan. 30, 2023 (GLOBE NEWSWIRE) -- scPharmaceuticals Inc. (Nasdaq: SCPH), a pharmaceutical...

Logo PR Newswire
HealthPress Release2023-01-31 07:45:00
HONG KONG, Jan. 31, 2023 /PRNewswire/ -- In response to the infection peak season of COVID-19 in rural areas that may be brought about by people returning...

Logo Benzinga
Business / FinanceBy Globe Newswire2023-01-31 06:00:00
The application includes data from the Phase 3 registration study of patients with resistant hypertension, where aprocitentan demonstrated a sustained blood...

Logo PR Newswire
HealthPress Release2023-01-30 12:00:00
DALLAS, Jan. 30, 2023 /PRNewswire/ -- Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative...

Logo EIN Presswire
HealthPress Release2023-01-31 10:05:00
Chagas disease can transmitted through blood transfusion, organ transplantation, as well as congenital and oral transmissions. BURLINGAME, CALIFORNIA, UNITED...

Logo GlobeNewswire
SciencePress Release2023-01-30 13:24:00
SHANGHAI, China, Jan. 30, 2023 (GLOBE NEWSWIRE) -- Shanghai Junshi Biosciences Co., Ltd (Junshi Biosciences, HKEX: 1877; SSE: 688180), a leading...

Logo PR Newswire
HealthPress Release2023-01-30 21:37:00
SILVER SPRING, Md., Jan. 30, 2023 /PRNewswire/ -- Today, the U.S. District Court for the Southern District of Florida entered a consent decree of permanent...

Logo Benzinga
Business / FinanceBy Pr Newswire2023-01-31 01:00:00
- Leveraging the success in Japan to expand the Alluminox platform globally and obtain new approvals as early as 2025 SAN DIEGO, Jan. 30, 2023 /PRNewswire/...