FDA Approves Spectrum Pharmaceuticals’ Orphan Cancer Drug BeleodaqBy
The US Food and Drug Administration (FDA) has approved Spectrum Pharmaceuticals’ Beleodaq (belinostat) for the treatment of patients with peripheral T-cell lymphoma (PTCL), a rare and fast-growing type of non-Hodgkin lymphoma (NHL). The action was taken under the agency's accelerated approval program.
PTCL comprises a diverse group of rare diseases in which lymph nodes become cancerous. In 2014, the National Cancer Institute estimates that 70,800 Americans will be diagnosed with NHL and 18,990 will die. Beleodaq works by stopping enzymes that contribute to T-cells, a type of immune cell, becoming cancerous. It is intended for patients whose disease returned after treatment (relapsed) or did not respond to previous treatment (refractory).
Beleodaq is the third drug that has been approved by FDA since 2009 for the treatment of peripheral T-cell lymphoma. The FDA granted accelerated approval to Folotyn (pralatrexate) in 2009 for use in patients with relapsed or refractory PTCL and Istodax (romidepsin) in 2011 for the treatment of PTCL in patients who received at least one prior therapy. Folotyn is also marketed by Spectrum Pharmaceuticals, and Istodax is marketed by Celgene Corporation.
The FDA's accelerated approval program allows for approval of a drug based on surrogate or intermediate endpoints reasonably likely to predict clinical benefit for patients with serious conditions with unmet medical needs. Drugs receiving accelerated approval are subject to confirmatory trials verifying clinical benefit. Beleodaq also received orphan product designation by the FDA because it is intended to treat a rare disease or condition.
Spectrum Pharmaceuticals, based in Hendersen, Nevada, is a biotechnology company focused on acquiring, developing, and commercializing drug products, with a primary focus in oncology and hematology. Spectrum and its affiliates market four oncology drugs: Fusilev (levoleucovorin) for injection in the US; Folotyn (pralatrexate injection), also marketed in the US; Zevalin (ibritumomab tiuxetan) injection for intravenous use, for which the company has worldwide marketing rights and Marqibo (vinCRIStine sulfate liposome injection) for intravenous infusion, for which the company has worldwide marketing rights.
Source: FDA and Spectrum Pharmaceuticals