Orphan Drugs: The Rising Stars
Thomson Reuters publishes a quarterly report, “The Ones to Watch”, that identifies and analyzes the “most promising drugs”, or what the Orphan Druganaut Blog calls “the rising stars”. The latest report published in September is for the Q2 2013, April – June 2013.
The review of the drugs are organized by the following four categories :
• Launched or receiving approval
• Entering Phase III trials
• Entering Phase II trials
• Entering Phase I trials.
The following drugs from the report identifies the most promising orphan drugs under these categories from the Q2 2013 :
Launched or Receiving Approval
Two orphan drugs in this category are from GlaxoSmithKline (GSK) and receive FDA approval in May 2013 for the treatment of metastatic melanoma :
• Mekinist (Trametinib)
• Tafinlar (Dabrafenib).
Both drugs are approved as single agents, not as a combination treatment. Tafinlar, a BRAF inhibitor, is approved for melanoma whose tumors express the BRAF V600E gene mutation. Mekinist, a MEK inhibitor, is approved for melanoma whose tumors express the BRAF V600E or V600K gene mutations. Both drugs are approved with a companion genetic test, ThxID BRAF, which will help determine if a patient’s melanoma cells have the V600E or V600K mutation in the BRAF gene. About 50% of melanomas arising in the skin have a BRAF gene mutation.
According to GSK’s Q3 2013 financial results :
• Mekinist and Tafinlar both launch in the United States late in the Q2 2013
• In the EU, Tafinlar receives approval in September 2013 and GSK files for regulatory approval of Mekinist in February 2013
• GSK files for regulatory approval of the combination, Trametinib + Dabrafenib, for metastatic melanoma in 2013 in both the United States and the EU, with FDA Priority Review given in September 2013.
Entering Phase II Trials
The orphan drug in this category, is Edimer Pharmaceuticals’ EDI200 for the potential treatment of the rare disease, X-linked Hypohidrotic Ectodermal Dysplasia (XLHED). The Cambridge, Massachusetts-based company, founded in 2009, is a biotechnology company focusing on therapeutics for rare genetic disorders. EDI200 has Orphan Drug Designation (ODD) and Fast Track status in the US, and ODD in Europe.
XLHED, also known as Christ-Siemens-Touraine Syndrome, is an ultra-rare genetic disease that causes symptoms including poor temperature control, respiratory issues, lack of sweat glands, and other problems. Patients affected by XLHED are at risk for life-threatening hyperthermia based on their inability to regulate body temperature, and for clinically significant pneumonias resulting from their abnormality in respiratory secretions. XLHED affects between one and nine individuals in every million.