An advisory panel at the U.S. Food and Drug Administration has rejected Alnara Pharmaceuticals’ drug candidate for cystic fibrosis. While the FDA as a whole does not have to accept the recommendation of the panel, it very often does. Cambridge, Mass.-based Alnara was bought by Eli Lilly and Co. last year.
The FDA panel - on a vote of 9 to 3 - determined that the clinical data for liprotamase, as it’s called, didn’t “constitute substantial evidence of the efficacy” of the drug, and said additional studies should be conducted.
More ominously, the panel also said, in a 7-to-4 vote, that the benefits of liprotamase didn’t outweigh the potential risks.
The news is a huge setback for a drug candidate that has been on a roller coaster ride for the past few years - and it is undoubtedly a tremendous disappointment for the Cystic Fibrosis foundation, which has invested millions in its development. The drug candidate was originally being developed by Altus Pharmaceuticals, which went under in 2009, a victim of the biotech cash crunch that followed the market crash in October of 2008. The CF foundation took control of the drug development’s for several weeks in the spring of 2009, until it was licensed by a new company, Alnara, which was led by some former Altus executives. The promise of liprotamase drove the sale of Alnara to Eli Lilly for $180 million, plus up to $200 million more in milestone payments, last summer.
Alnara CEO Alexey Margolin said in an interview a few months ago, after the drug was submitted to the FDA, “I hope for a Hollywood ending to this story.”
The FDA is expected to make a final decision in April.
The FDA panel - on a vote of 9 to 3 - determined that the clinical data for liprotamase, as it’s called, didn’t “constitute substantial evidence of the efficacy” of the drug, and said additional studies should be conducted.
More ominously, the panel also said, in a 7-to-4 vote, that the benefits of liprotamase didn’t outweigh the potential risks.
The news is a huge setback for a drug candidate that has been on a roller coaster ride for the past few years - and it is undoubtedly a tremendous disappointment for the Cystic Fibrosis foundation, which has invested millions in its development. The drug candidate was originally being developed by Altus Pharmaceuticals, which went under in 2009, a victim of the biotech cash crunch that followed the market crash in October of 2008. The CF foundation took control of the drug development’s for several weeks in the spring of 2009, until it was licensed by a new company, Alnara, which was led by some former Altus executives. The promise of liprotamase drove the sale of Alnara to Eli Lilly for $180 million, plus up to $200 million more in milestone payments, last summer.
Alnara CEO Alexey Margolin said in an interview a few months ago, after the drug was submitted to the FDA, “I hope for a Hollywood ending to this story.”
The FDA is expected to make a final decision in April.