Complete response, priority review and orphan drug designations.
Tivozanib (Aveo) for the treatment of patients with advanced renal cell carcinoma (RCC). In the CRL, FDA stated that the post-study treatment data for tivozanib produced inconsistent progression-free survival and overall survival (OS) results, thus making the TIVO-1 results uninterpretable and inconclusive. FDA recommends that the company conduct an additional clinical study to support approval of tivozanib for the treatment of advanced RCC. In addition, FDA also stated that the proposed dissolution acceptance criterion was not supported by the provided dissolution data, and would need to be updated and resubmitted.
Testosterone undecanoate (AVEED, Endo Pharmaceuticals) injection, for men diagnosed with hypogonadism. The complete response letter did not include requests for the company to perform additional clinical studies. FDA outlined the steps necessary to support approval of the New Drug Application and updated the requirement for a Risk Evaluation and Mitigation Strategy (REMS). Specifically, FDA has requested that the REMS include a Medication Guide as well as Elements to Assure Safe Use (ETASU) to mitigate the risks and severe complications related to post-injection reactions. Endo plans to submit a complete response by the end of the third quarter of 2013.
Metreleptin (AstraZeneca and Bristol-Myers Squibb) for the treatment of metabolic disorders associated with inherited or acquired lipodystrophy.
Sofosbuvir (Gilead Sciences), a once-daily oral nucleotide analogue inhibitor for the treatment of chronic hepatitis C virus (HCV) infection.
Miltefosine (Impavido, Paladin Labs) for the treatment of leishmaniasis, one of the diseases targeted by FDA for innovation and development of new therapies through its tropical disease priority review voucher program.
Orphan drug designations
SL-401 (Stemline Therapeutics) for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and aggressive hematologic malignancy for which there is no effective treatment.
RV001 (River Vision Development), a human monoclonal antibody teprotumumab, for the treatment of active phase Graves Orbitopathy (GO), also known as thyroid eye disease.