Alnylam Selects PANTHERx to Distribute its Recently Approved AHP Treatment Givlaari

Alnylam Selects PANTHERx to Distribute its Recently Approved AHP Treatment Givlaari
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Alnylam Pharmaceuticals has tapped PANTHERx Rare Pharmacy to be a limited distribution partner for its newly approved therapy Givlaari (givosiran) to treat acute hepatic porphyria (AHP).

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.

 

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”
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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
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Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”
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