Akcea Therapeutics Inc. (NASDAQ: AKCA) shares saw a nice bump on Wednesday after the company announced in conjunction with Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) that they had received a marketing authorization approval from the European Commission (EC).
Specifically, this approval is for the treatment, Tegsedi, of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). This follows the positive opinion recommending approval provided by the Committee for Medicinal Products for Human Use (CHMP) of European Medicines Agency (EMA).
The EC’s approval of Tegsedi was based on results from the Phase 3 NEURO-TTR study in patients with hATTR with symptoms of polyneuropathy. Results from that study demonstrated that patients treated with Tegsedi experienced significant benefit compared to patients treated with placebo.
Paula Soteropoulos, CEO of Akcea, commented:
With the EC`s decision, TEGSEDI is now the world`s first and only RNA-targeted therapeutic approved for patients with hATTR amyloidosis. With subcutaneous delivery, TEGSEDI puts treatment in the patients` hands while bringing the significant benefits shown in the NEURO-TTR study in both measures of neuropathy and quality of life for people living with this serious and fatal disease. This is an important day for the hATTR amyloidosis community as we believe TEGSEDI enables people and their families impacted by this disease to move forward with their lives. Today is a milestone for Akcea with our first drug approval. It is an achievement we share with the courageous hATTR patient community in Europe and around the globe. We are ready to launch TEGSEDI along with our patient and physician support services across Europe.
Shares of Akcea were last seen up about 3% at $24.96, with a consensus analyst price target of $32.00 and a 52-week trading range of $1.05 to $33.99.
Ionis shares were trading at $45.00, with a consensus price target of $58.50 and a 52-week range of $39.07 to $65.51.