March 17, 2017
By Alex Keown, BioSpace.com Breaking News Staff
SAINT-GENIS-POUILLY, France – Despite technical issues that resulted in a Complete Response Letter for French biotech firm Advanced Accelerator Applications (AAA), Chief Executive Stefano Buono believes cancer drug Lutathera represents a “multi-hundred million dollar opportunity.”
In an interview with Fortune, Buono said he believes the U.S. Food and Drug Administration will approve commercialization of the drug that targets gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut neuroendocrine tumors in adults. Lutathera is a Lu-177-labeled somatostatin analogue peptide. The drug belongs to an emerging form of treatments called Peptide Receptor Radionuclide Therapy (PRRT), which involves targeting neuroendocrine tumors with radiolabeled somatostatin analogue peptides.
During Phase III results, the drug showed promise in staving off death in patients. Data showed the drug demonstrated a statistically significant and clinically meaningful risk reduction of 79 percent of disease progression or death versus Octreotide LAR 60. The data was so convincing that approximately 1,600 cancer patients in France have been treated with the drug under a compassionate use program. In that country, Lutathera has been granted a temporary authorization of use. As a result, AAA has set an initial price of about $68,600 for four treatments with the drug, Fortune reported. The type of cancerous cells Lutathera targets are the same kind that ultimately lead to the death of Steve Jobs. Jobs, the founder of Apple , died of tumors originating in the endocrine glands which are among the rarer forms of pancreatic cancer.
While it is being used in France, the drug has had a rocky time in the United States. In December, the company received a Complete Response Letter that outlines problems with the filing of data regarding the drug. In the letter, the FDA pointed to issues with the “format, traceability, uniformity, and completeness relating to the NETTER-1 and Erasmus clinical datasets,” the company said. Additionally AAA said the CRL called for subgroup analyses based on gender, age and racial subgroups. The regulatory agency also requested a safety update on clinical and non-clinical studies. In December, Buono said the company was addressing the issues highlighted in the CRL and was planning to resubmit the data to the FDA.
Buono told Fortune that AAA anticipates refiling with the FDA toward the end of 2017. The company will also seek regulatory approval in Europe this year as well, he said. Shares of AAA are up slightly this morning, trading at $36.84.
