Enforcement Report - Week of July 6, 2022
For $200 million upfront, AstraZeneca is licensing Ionis’ eplontersen, the California biotech’s second shot at the rare disease transthyretin amyloidosis (ATTR) after a rather lackluster performance by its predecessor Tegsedi. Up to $485 million in development and approval milestones, plus another $2.9 billion payment tied to sales, are up for grabs.
SOUTH PLAINFIELD, N.J., Oct. 11, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Tegsedi® (inotersen) has successfully received Category 1 classification from Câmara de Regulação do Mercado de Medicamentos - CMED (Drug Market Regulation Chamber) in Brazil. Category 1 classification is given to innovative treatments that provide greater efficacy than current standards of care. Category 1 allows for pricing in line with international markets.
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.
BOSTON, July 22, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., today announced that the Main Association of Austrian Social Security Institutions (Dachverband der Österreichischen Sozialversicherungsträger, or DV) has granted approval for the national reimbursement of TEGSEDI® (inotersen) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin (hATTR) amyloidosis.
BOSTON, June 8, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., today announced that AIFA, or The Italian Medicines Agency, has granted approval for the reimbursement of TEGSEDI® (inotersen) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin (hATTR) amyloidosis.
BOSTON, June 1, 2020 /PRNewswire/ -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., today announced the Portuguese Medicine Regulatory Authority (INFARMED) has granted approval for reimbursement of TEGSEDI® (inotersen) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin (hATTR) amyloidosis.
BOSTON and CARLSBAD, Calif., May 28, 2020 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), today announced publication of long-term data from the open-label extension (OLE) of the pivotal NEURO-TTR study of TEGSEDI® (inotersen) in patients with hereditary transthyretin (hATTR) amyloidosis with polyneuropathy. The primary objective of the OLE study was to evaluate the safety and tolerability of long-term dosing with TEGSEDI. Secondary objectives of the study included understanding progression based on measures such as the modified Neuropathy Impairment Score +7 (mNIS+7) and the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN). Understanding changes over time in generic health-related quality of life based on the Short Form 36 Health Survey (SF-36) was an exploratory objective. This interim analysis, published in the European Journal of Neurology, show that treatment with TEGSEDI was not associated with additional safety concerns or signs of increased toxicity in study participants treated for up to five years. Treatment with TEGSEDI resulted in continued efficacy in patients after two years. Results also showed that patients who started treatment with TEGSEDI earlier (received TEGSEDI treatment in the NEURO-TTR study) achieved greater long-term disease stabilization compared to those who switched from placebo to TEGSEDI in the OLE study. For the full text of this publication, please visit: https://onlinelibrary.wiley.com/doi/abs/10.1111/ene.14285
OTTAWA, le 13 mai 2020 /CNW/ - Akcea Therapeutics Canada, Inc., une société affiliée d'Akcea Therapeutics, Inc. (NASDAQ : AKCA), a annoncé aujourd'hui la fin de ses négociations avec l'Alliance pancanadienne pharmaceutique (APP), avec la signature d'une lettre d'intention concernant TEGSEDIMC (inotersen), un médicament indiqué pour le traitement de polyneuropathie de stade 1 ou 2 chez des patients adultes avec amylose héréditaire de la transthyrétine (ATTR héréditaire)1. Akcea Therapeutics, Inc. est une société affiliée détenue majoritairement par Ionis Pharmaceuticals, Inc.
As Akcea Therapeutics get rolling with new rare-disease med Waylivra, it's filling a new post. Its first commercial chief is joining the C-suite from inside the company.