According to a report by biopharmadive.com, Pfizer announced they will be dropping support for an experimental Duchenne muscular dystrophy treatment. The company intends to see if the treatment may be useful in other areas, but based on trial results will be stepping away from it in regards to Duchenne. Keep reading to learn more or follow the original story here for more information.
What is Duchenne Muscular Dystrophy?
Duchenne muscular dystrophy, also referred to as DMD or Duchenne, is one of nine forms of muscular dystrophy. People living with Duchenne muscular dystrophy typically experience muscle weakness as a result of their body being unable to produce dystrophin. This may eventually lead to difficulties with the heart, and lungs, as well as quadriplegia.
Duchenne muscular dystrophy is an inherited condition passed by an x-linked recessive pattern. On a global scale, Duchenne muscular dystrophy affects one in every 3,500 male births and one in about every 50 million female births.
Rare disease treatments have become an area of focus for drugmakers in recent years. Patient populations may be small but the money is not. Pfizer made $1.12 billion just through rare diseases in the first half of 2018. With only one FDA approved Duchenne muscular dystrophy drug in existence, Pfizer saw an opportunity to step in.
Pfizer began investigating three different options for Duchenne. One of which is known as domagrozumab. The drug was found to provide no significant benefits to patients after clinical trials. The drug did not even outperform the placebo in one trial. The decision to drop domagrozumab means that an ongoing Phase 2 trial will be stopped. The company may, however, continue to pursue its other options for Duchenne.
Pfizer’s main other treatment that appears to be making progress on DMD is a form of gene therapy. Pfizer’s CEO maintains that the company invested substantial sums in this treatment and will continue to look for opportunities to use what they learned through it or create some form of deal. This information was revealed during an earnings call. It was also expressed that the company would be looking commit to the area of research, but won’t buy anything overvalued.