The medication was approved in November by the U.S. Food and Drug Administration (FDA) for adults with AHP, a group of rare diseases caused by mutations in genes responsible for the production of enzymes that are involved in the heme biosynthesis pathway. Of note, heme is a key molecule in many cellular processes, including oxygen transport.
“PANTHERx is proud to deliver innovative medicine to adults with acute hepatic porphyria,” said Gordon Vanscoy, PANTHERx chairman and CEO, in a press release. “As a proud partner with Alnylam, we are appreciative of the opportunity to bring new hope to a population of patients who previously had limited therapeutic options. We are thrilled to have a partner whose mission so closely aligns with our own as we collaborate to deliver a new treatment to this patient community.”
Developed by Alnylam, givosiran is an investigational RNA-based treatment designed to impede activity of the aminolevulinic acid (ALA) synthase 1 enzyme, preventing the AHP-causing accumulation of toxic molecules. The medication can prevent or reduce the occurrence of severe and life-threatening attacks, and ease AHP symptoms. (RNA is a DNA-generated molecule that guides production of a specific protein).
The treatment’s approval last month came less than four months after the FDA accepted the therapy’s new drug application, which was granted priority review status. It was based on positive results of the Phase 3 ENVISION study (NCT03338816), which measured the safety and effectiveness of givosiran in reducing disease attacks.
The study’s final data indicated that, compared with a placebo, givosiran lowered patients’ annual rate of attacks by 74%. The treatment also reduced levels of ALA — a key AHP biomarker — in patients’ urine by 92%.
AHP can be accompanied by neurovisceral attacks in the form of intense abdominal pain, muscle weakness, sensory loss, urination problems, convulsions, irritability, anxiety, breathing problems, hallucinations, and mental confusion. Long-term complications can include chronic neuropathic pain, hypertension, liver disease, and chronic kidney disease.
PANTHERx, based in Pittsburgh, Pennsylvania, was named 2018 Specialty Pharmacy of the Year by the National Association of Specialty Pharmacy, and is reportedly the sole U.S. health entity with an accredited distinction in rare pharmacy. The company works to streamline the process of getting rare medications to patients.
Last month, Alnylam said it is working with payers to accelerate patients’ access to Givlaari, and that it had reached a pact in principle with Harvard Pilgrim to cover the treatment. Commercially insured patients are expected to have little to no out-of-pocket medication costs.