First potential Prader-Willi treatment among EMA’s orphan recommendations

Ferring’s carbetocin, potentially the first treatment for Prader-Willi syndrome, is among 16 new medicines recommended to receive orphan drug designation in Europe.

The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) said work on carbetocin’s use to treat the rare genetic disorder should be granted development incentives.

Prader-Willi syndrome, which is estimated to affect less than 1 in 5,000 people in the EU, causes a variety of symptoms, including a constant desire to eat food, leading to obesity, impaired function of the gonads, learning difficulties and behavioural problems.

Patients with the disease often have a significantly reduced life span and require lifelong care.

Carbetocin’s recommendation was supported by the European Organisation for Rare Diseases (Eurordis), a European alliance of patient organisations and individuals involved in the promotion of research for rare diseases and development of orphan drugs.

Other treatments to be recommended by the COMP for orphan drug designation, which is granted to products for life-threatening or very serious conditions that affect no more than 5 in 10,000 people in the EU, included Nexus Oncology’s diamidophosphate.

The drug, which is being investigated as a treatment for soft tissue sarcoma, was joined on the list by TMC Pharma Service’s chlormethine for treatment of cutaneous T-cell lymphoma and Laboratoire HRA Pharma’s ketoconazole for treatment of Cushing's syndrome.

Therapies for neglected tropical diseases also featured in the COMP’s recommendations, with Dafra Pharma’s oleylphosphocholine intended to treat leishmaniasis – a disease caused by sand fly parasites that is estimated to cause as many as 50,000 deaths per year.

The EMA said that orphan medicine incentives can be also used to support the development of treatments for similar neglected diseases, which would not be developed under normal market conditions.

All the COMP’s recommendations are now with the European Commission, which will make the final decision on each drug.

The full list of EMA orphan drug recommendations

From http://www.pmlive.com/pharma_news/potential_first_prader-willi_treatment_ema_orphan_recommendation_357835

The newest book in the Cushie Bookstore

Alone in My Universe: Struggling with an Orphan Disease in an Unsympathetic World

Battling a chronic disease can often be a lonely and frustrating ordeal. It doesn’t have to be. Alone in My Universe, compiled by Wayne Brown, presents a series of writings from patients suffering from acromegaly. Based on actual personal experiences, these narratives provide a primer of understanding for others dealing with this chronic, debilitating disease that affects many of the body’s systems. This collaborative effort, written by real people touched by acromegaly, attempts to raise awareness of this low-profile disease. Each patient tells his or her story as it relates to the disease and its complications.

Topics include the following:

• Handling pre-diagnosis frustration

• Dealing with fears of medical treatment and surgery

• Managing family issues and work issues

• Talking with children

• Managing negative energy

• Coping with good and bad days

• Living life each day With sympathy, empathy, and mutual support, Alone in My Universe shares the heartfelt stories of those suffering from acromegaly to show others that they are not alone in their battle.

MaryO'Note: This book is great for anyone with any orphan disease - it's not only for acromegaliacs!