Orphan Status for Allos Drug in EU - Analyst Blog

Recently, Allos Therapeutics (ALTH) received orphan drug status from the European Commission (EC) for Folotyn (pralatrexate) for the treatment of cutaneous T-cell lymphoma (CTCL). The candidate is being evaluated in an early-stage study to treat patients suffering from relapsed or refractory CTCL. 

Furthermore, Allos intends to conduct a late-stage study to evaluate the candidate in combination with systemic bexarotene versus only systemic bexarotene in CTCL patients who have not responded to at least one prior systemic therapy. However, before initiating the study, the company intends to carry out an early-stage trial to determine the maximum tolerated dose of the combination. The early-stage study is expected to commence in 2010. 

CTCL comprises approximately 2% to 3% of the estimated 66,000 new cases of non-Hodgkin's lymphoma diagnosed each year in the US, according to data released by the Lymphoma Research Foundation. CTCLs comprise a group of sluggish non-Hodgkin's T-cell lymphomas, which mainly manifest in the skin. 

The orphan drug designation brings with it ten years of marketing exclusivity subsequent to the candidate getting marketing approval in the European Union. Additionally, this status, which is based on a favorable opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), provides several other benefits such as free protocol assistance on clinical trials, a reduced Marketing Authorisation Application (MAA) filing fee and the possibility for grant funding. Furthermore, the designation facilitates the development of drugs that may provide significant benefit to patients suffering from rare, life-threatening diseases.
 
Folotyn already enjoys orphan drug status from the EC for other indications such as peripheral T-cell lymphoma (PTCL) and non-papillary transitional cell carcinoma (TCC) of the urinary bladder, a form of bladder cancer. Folotyn is currently approved by the US Food and Drug Administration (FDA) for treating patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). The drug received accelerated FDA approval in September 2009 and is the only marketed product at Allos. 

We believe Folotyn’s approval for the PTCL indication will provide major relief to patients suffering from this disease as prior to Folotyn there was no approved drug. While the drug has been available to patients since October, it was launched commercially in January 2010. 

The company’s efforts to expand Folotyn’s label into other indications is encouraging. We believe that Folotyn, if approved for any of the additional indications can prove to be a major revenue earner for the company. 

Currently we are Neutral on Allos Therapeutics.
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