Akcea Therapeutics genetic disease treatment gets FDA approval

Akcea Therapeutics Inc said on Friday its treatment, developed along with Ionis Pharmaceuticals , which targets a rare genetic disease was approved by the U.S. Food and Drug Administration.

Akcea’s once-weekly injection, Tegsedi, treats hereditary ATTR amyloidosis patients with polyneuropathy.

Polyneuropathy, a symptom of the disease, is the simultaneous malfunction of peripheral nerves in the body resulting in tingling, numbness and kidney dysfunction.

Tegsedi belongs to a class of drugs that uses the Nobel-prize winning RNA interference mechanism to manipulate ribonucleic acid, which interferes with or “silences” targeted genes and stops the formation of proteins that can cause diseases.

Tegsedi’s approval in the United States comes nearly three months after its approval in Europe.

Based on the U.S. approval of Tegsedi, Ionis will receive a $50 million milestone payment and both the companies will split the gains from the drug’s sale at a ratio of 60 to 40 in favor of Ionis, Akcea said.

Earlier this year, the drug regulator had declined to approve Akcea’s similar drug, Waylivra, which looked to treat a genetic disease causing fat accumulation in the blood.

  • Related Posts

    • Pharma
    • July 22, 2025
    • 149 views
    DGFT Extends Deadline on Application for Export of Pharma Grade Sugar

    The Directorate General of Foreign Trade ( DGFT ) has notified an extension in the deadline for submitting applications for the export of Pharma Grade Sugar. This was communicated through Trade Notice…

    • Pharma
    • July 16, 2025
    • 577 views
    Aurobindo, Cipla, Viatris to make, market long-acting injectable HIV drug

    Generic drugmakers Aurobindo Pharma, Cipla and Viatris will develop, manufacture and supply long-acting injectable cabotegravir (CAB LA) for HIV treatment in 133 countries. This follows ViiV Healthcare, a company focused…

    Leave a Reply

    Your email address will not be published. Required fields are marked *

    You Missed

    10, 000 Km is just a Number for Intercontinental Tele Surgery by SSIMantra

    10, 000 Km is just a Number for Intercontinental Tele Surgery by SSIMantra

    DGFT Extends Deadline on Application for Export of Pharma Grade Sugar

    DGFT Extends Deadline on Application for Export of Pharma Grade Sugar

    Bihar Healthcare is poised to be embraced by Robotic Surgery Mantra

    Bihar Healthcare is poised to be embraced by Robotic Surgery Mantra

    Aurobindo, Cipla, Viatris to make, market long-acting injectable HIV drug

    Aurobindo, Cipla, Viatris to make, market long-acting injectable HIV drug