safety of the oral drug and compare the growth, height, weight, and vitamin absorption of CF patients with what are considered normal levels among healthy individuals, Gallotto says. The trial studied the effects of the drug in each patient over a 12-month period. When Altus was still developing the drug late last year, it reported positive results of an initial late-stage clinical trial to study its effects in CF patients. But this latest study was needed to seek approval of the drug.
Alnara is already in talks with the FDA about the trial results and is writing an application for approval of liprotamase, aiming to submit a New Drug Application (NDA) to regulators by the end of the year, Gallotto says. The company—which launched last year with a $20 million Series A round of venture capital—has enough funding to support it up until it submits the NDA, he adds.
Liprotamase is designed to offer more stable enzymes than existing treatments and may enable patients to take fewer pills than they do with the other enzyme drugs. Liprotamase is also made from engineered microbes, as compared with versions of the drug now on the market derived from pancreatic enzymes in pigs. Gallotto says that regulators have noted concerns about the potential risk of those other treatments carrying swine viruses. Still, the FDA earlier this month granted formal approval to one of these drugs, marketed by Solvay Pharmaceuticals under the brand name Creon, saying that the treatment met safety and efficacy standards.
The U.S. market for such enzyme therapies is about $300 million annually, Gallotto says. Alnara hopes to begin marketing liprotamase in the U.S. next year.
