After rocky hep C path, Achillion finds surer footing with FDA breakthrough status
Achillion’s promising proof-of-concept data for its latest drug has won breakthrough status at the FDA.
The US regulator granted its coveted designation to danicopan as a treatment for anemia in paroxysmal nocturnal hemoglobinuria patients taking the mainline C5 inhibitor treatment Soliris. Up to 75% of PNH patients suffer from anemia even after treatment, according to Achillion, with up to a third requiring blood transfusions in the past year.
The news comes as a boon for Achillion $ACHN, still reeling from its decision last year to shelve a drug they hoped could compete as a monotherapy for PNH patients. The company’s stock shot up 14%. In Q2 the biotech reported losses close to $20 million, leaving $241 million in cash and securities in reserve.
The breakthrough designation was based on interim data from a Phase II trial, and the biotech said they hope to have full results by the end of 2019 with a Phase III trial underway by early 2020.
Achillion presented the Phase II data from 11 patients at a May conference. They found patients’ hemoglobin increased by 2 g/dl at week 4, with average increases of 2.6 g/dl for patients who reached week 24. This meant a dramatic decrease in blood transfusions for patients – from 34 transfusions totaling 54 units to one transfusion totaling two units.
Patients also improved on a functional assessment of chronic illness therapy fatigue versus baseline, increasing on average from 4 t0 11. Achillion reported the drug was “generally well tolerated” when added to Soliris.
Achillion originally found the spotlight as a prime contender in the hepatitis C field, until Gilead came up with its painless cure and dramatically shrunk the market. That prompted a pivot to ACH-4471 as their primary target. After some rocky riding, it seems to have found a footing.
Alexion has raked billions from Soliris, both for treating PNH and other blood disorders such as atypical hemolytic uremic syndrome, but now faces potential competition in the form of a biosimilar from Amgen.
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