News

Sarepta Therapeutcis, a medical research and drug development company, has announced the approval of Elevidys, its gene therapy for Duchenne’s muscular dystrophy (DMD), by United Stated Food and Drug Administration (FDA). Elevidys is the first gene therapy approved for the treatment of DMD.

DMD is an X-linked, degenerative muscle disease generally manifesting in early childhood, eventually leading to effective paralysis due to muscle weakness. The life expectancy is around 26-years of age and there is currently no curative therapy available.

Elevidys (delandistrogene moxeparvovec-rokl) consists of modified adeno-associated virus (AAV) particles of serotype rh74, carrying a gene coding for micro-dystrophin, which is a shortened version of the dystrophin gene mutated in DMD patients. The intravenous infusion at the dose of 1.33 x 10^14 vector genomes/kg is a one-time treatment, with current data supporting effectiveness at least up to 4 years (SRP-9001-103).

Read more.

HEMGENIX^® (etranacogene dezaparvovec) has received a conditional marketing authorisation from the European Commission.

UniQure, a company specialising in the development of gene therapies, has announced that the gene therapy Hemgenix, developed by uniQure and licensed by CSL Behring, has been approved in Europe for the treatment of severe and moderately severe haemophilia B.

Haemophilia B is a bleeding disorder caused by the lack of coagulation factor IX (FIX). It is perhaps best known for its influences in the European royal houses, such as in the severe case of Aleksei Romanov, heir to Nikolai II of Russia. Indeed, apart from very mild cases haemophilia B is almost solely seen in men due to the FIX gene being located to the X-chromosome, with women generally carrying a compensating healthy copy on their second X-chromosome.

In addition to external bleeds from cuts and wounds the patients suffer from internal bleeds for example to joints, which is extremely painful and can cause lasting damage. Haemophilia B is treated with intravenous injections of functional FIX, with patients suffering from the severe form sometimes requiring injections up to several times a week.

Hemgenix uses modified adeno-associated virus of serotype 5 (AAV5) to deliver a functional gene copy of FIX into the patient cells. The cells then read the instructions for making FIX in the delivered DNA and secrete the functional coagulation factor into the blood. Hemgenix is a one-time treatment, which in the clinical trials has demonstrated significantly reduced bleeds and eliminated the need for routine Factor IX injections for almost all patients.

Fimea has started the assessment of the therapeutic and economic value of Hemgenix, which will contribute to determining whether it will be offered by the public healthcare system in Finland.

Lähteet: uniQure, FIMEA, Duodecim

Ferring Pharmaceuticals has announced that the novel gene therapy for bladder cancer developed by its sister company FinVector Oy has been approved by FDA. FinVector Oy has developed gene medicines in Kuopio for 30 years, and the development of gene therapy for bladder cancer was also already started in the 1990s.

Adstiladrin® (nadofaragene firadenovec-vncg) contains modified adenoviruses carrying a human gene that stimulates immune responses (IFNα). The medicine works by entering and killing cancer cells, which then further stimulates the patient’s own immune system to kill more cancer cells.

Read more on the FinVector website.