PTC Therapeutics has received approval from the Brazilian health regulatory agency, ANVISA, for Waylivra (volanesorsen) to treat familial partial lipodystrophy (FPL).
Enforcement Report - Week of July 6, 2022
BOSTON, Sept. 23, 2021 /PRNewswire/ -- BIORCHESTRA is a bio-tech company focused on the identification of novel targets in neurodegenerative diseases using RNA-based therapeutics. To access these novel targets, BIOCHESRTRA has also developed a proprietary nanoparticle formulation to deliver RNA-based drugs across the blood-brain barrier in sufficient amounts to be therapeutically effective. In addition, their proprietary exosome-based diagnostic that detects and quantifies markers of neuroinflammation and neurodegeneration facilitates patient selection and treatment monitoring.
Ionis Pharmaceuticals will lay off nearly 70% of the workforce at Akcea Therapeutics, the spinout it formed in 2014 to market some of its medicines but then acquired and folded back in last year.
About half a year after reabsorbing its lipid-focused spinout Akcea, Ionis is now putting a major reorganization plan in play — one that involves shaving nearly 70% of the subsidiary’s workforce and expanding its distribution deal with Sobi.
LONDON--(BUSINESS WIRE)--Akcea Therapeutics, a wholly owned subsidiary of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced today that the Scottish Medicines Consortium (SMC) has enabled access to volanesorsen on the National Health Service (NHS) in Scotland for the treatment of familial chylomicronaemia syndrome (FCS).1 This decision will allow patients in Scotland with this rare and life-threatening condition to access the treatment through NHS Scotland. SMC’s acceptance follows recent approval from the National Institute for Health and Care Excellence (NICE) for the use of volanesorsen on the NHS in England.2
BOSTON & CARLSBAD, Calif.--(BUSINESS WIRE)--Ionis Pharmaceuticals (NASDAQ: IONS) and its wholly owned subsidiary, Akcea Therapeutics, today recognize the 3rd annual Familial Chylomicronemia Syndrome (FCS) Awareness Day, a global observance held each year on the first Friday in November to build broader awareness of the challenges associated with this debilitating, ultra-rare disease. Together with members of the FCS community, Ionis and Akcea have made significant progress in efforts to help people around the world learn about FCS and the impact it has on patients, their families and caregivers.
CARLSBAD, Calif. and BOSTON, Nov. 3, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) and its wholly owned subsidiary Akcea Therapeutics, Inc., today announced that Pfizer Inc. (NYSE: PFE) has initiated a Phase 2b study of vupanorsen (AKCEA-ANGPTL3-LRx) in statin-treated patients with elevated non-high-density lipoprotein cholesterol (non-HDL-C) and triglycerides (TGs). The study, Targeting ANGPTL3 with an antisense oligonucleotide in adults with dyslipidemia (TRANSLATE-TIMI 70), will evaluate various doses of vupanorsen to inform potential future development.
CARLSBAD, Calif. and BOSTON, Nov. 3, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) and its wholly owned subsidiary Akcea Therapeutics, Inc., today announced that Pfizer Inc. (NYSE: PFE) has initiated a Phase 2b study of vupanorsen (AKCEA-ANGPTL3-LRx) in statin-treated patients with elevated non-high-density lipoprotein cholesterol (non-HDL-C) and triglycerides (TGs). The study, Targeting ANGPTL3 with an antisense oligonucleotide in adults with dyslipidemia (TRANSLATE-TIMI 70), will evaluate various doses of vupanorsen to inform potential future development.
BOSTON and CARLSBAD, Calif., Oct. 30, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) and its wholly owned subsidiary, Akcea Therapeutics, received the Prix Galien USA Award for the Best Biotechnology Product in 2020 in recognition of TEGSEDI, the first and only self-administered, subcutaneous treatment for the polyneuropathy of hereditary ATTR amyloidosis in adults. This Prix Galien award recognizes outstanding achievements in biomedicine that improve the human condition and was presented at a virtual ceremony on Thursday, Oct. 29.