Orphan Drugs to Watch This Quarter

In Clinical Development by Cameron

Thomson Reuters has issued it’s Ones to Watch for Q2 2014 and once again a number of orphan products have made the short list. Whilst the report does not trumpet the success of the orphan drug act as much as their last release, there are still 3 orphan drugs that have made the list.

Already launched or Receiving Approval

Alprolix – Hemophillia B – Biogen Idec

Alprolix is a iv formulation of a long-acting, recombinant fusion protein comprising factor IX linked to the amino terminus of the Fc domain of IgG1 for the treatment of Hemophillia B. The treatmnent has been shown to control upto 90% of bleeding episodes with just one infusion every 10 days (down from 2-3 a week)

World wide sales are expected to reach $121m (2015), £178m (2016), $216m (2017).

Haemophilia B is an x-linked recessive disorder that prevents the blood from clotting due to a deficiency in factor IX. Haemophilia B is the second most common form of Haemophilia, and affects around one-in-25,000 men. The severity of Haemophilia cases can vary, but for sufferers of sever haemophilia patients undergo prophylactic infusions two or three times a week to prevent bleeding episodes. Alprolix is the first treatment that maintains prolonged circulation of factor IX concentrates in the body which could reduce the need for prophylactic infusions down to once every ten days.

Aprolix has recieved both Orphan Drug status and a Fast Track designation from the FDA, and approval in both the US and Canada.

Defitelio – Severe Hepatic Veno-occlusive disease – Gentium

Gentium, part of Jazz Pharmaceuticals, began to roll out Deitelio across Europe at the end of March following approval in October 2013. Defitelio is a treatment for severe hepatic veno-occlusive disease (VOD) in children and adults undergoing hematopoietic stem cell transplantation therapy.

This is the first therapy to be approved for the treatment of severe VOD in Europe where it is classified as a rare disease. the drug demonstrated a 52% increase in patient survival when compared with standard supportive care.

The drug has been approved under exceptional circumstances, and as such Gentium are required to create a pateint registry and provide long-term safety and outcomes to the EMA.

Entering Phase III trials

BMN-701 – Pompe Disease – BioMarin Pharmaceutical

BioMarin’s BMN-701, a replacement enzyme therapy for late onset Pompe disease, is moving towards PIII after positive phase II trials demonstrating substantially improved outcomes per dose over Genzyme’s Lumizyme.

Pompe disease is progressive degenerative disease caused by a deficiency in the lysomal enzyme acid-alpha glucosidase that leads to a build up of glycogen which leads to cell death. This affects heart and skeletal muscle causing respiratory problems and the enlarging of liver and heart. Adult onset Pompe affects about 1-in-57,000.

Lumizyme currently monopolises the market for Pompe treatments and so BMN-701’s impressive outcomes could make it an instant hit. Reuters predicts that BMN-701 will produce sales of $229 million by 2019.

The treatment is a fusion protein consisting of recombinant human insulin-like growth factor 2 fused to acid-alpha-glucosidase (GAA).