Amryt Pharma PLC (NASDAQ:AMYT, LON:AMYT) said it was ‘encouraged’ by data from an investigator-sponsored study of lomitapide in Familial Chylomicronaemia Syndrome (FCS).
Expert lipidologists in Italy administered the drug, also known as Juxtapid, or Lojuxta, to 18 people with the genetic disease, which results in elevated triglyceride levels, and brings with it “significant risk and disease burden”.
The 26-week, open-label assessment found there had been a 70.5% median triglycerides reduction versus the baseline. Lomitapide was generally well-tolerated, Amryt said.
The company said it will now “evaluate and analyse” the full data set, adding that it plans to discuss the potential development path with the Food & Drug Administration in the US and the European Medicines Agency.
Amryt’s chief medical officer, Dr Mark Sumeray, told investors: “We are encouraged by the data from the study and look forward to our discussions with the respective health authorities to progress lomitapide as a potential treatment for FCS.”