EMA Panel Backs Givlaari, First Treatment for Acute Hepatic Porphyria

Troy Brown

January 31, 2020

The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended marketing authorization for givosiran (Givlaari, Alnylam), the first treatment for acute hepatic porphyria (AHP) for patients aged 12 years and older.

"Acute hepatic porphyria is a rare genetic condition in which patients lack certain enzymes needed to produce heme, a basic structure of hemoglobin that binds to oxygen and is characterized by an accumulation of porphyrins in the body to toxic amounts," according to an EMA news release.

Patients with AHP can experience severe abdominal pain, vomiting, and nervous system disorders, including seizures, depression, and anxiety. The disorder is potentially fatal as a result of the potential for paralysis and respiratory arrest during disease exacerbations.

The US Food and Drug Administration (FDA) approved givosiran in November. Reuters reports that the drug has been priced in the US at $575,000 per year, not including discounts.

The drug's active substance, givosiran, consists of a "short, synthetic strand of genetic material called 'small interfering RNA' that has been designed to interfere with the production of an enzyme involved in an early step in making haem," according to the news release.

Blocking this action should prevent the production of substances that build up in the body and cause the disease's symptoms.

The committee's opinion follows consideration of safety and efficacy data from a phase 3 clinical trial that included 94 patients with AHP who experienced two or more attacks during the past 6 months. Patients who received givosiran experienced significantly decreased annual attacks, reduced pain, and better quality of life.

There are currently no approved treatments that directly relieve or prevent chronic symptoms that many patients experience, nor approved medications to lower the risk for attacks. The only treatment currently approved for treatment of acute AHP attacks is intravenous hemin, a heme formulation derived from human blood; however, it is not approved for use as a chronic therapy to prevent attacks.

Other treatments include analgesics and antiemetics, chemically-induced menopause with hormonal suppression therapy, and liver transplantation.

Givosiran addresses an unmet medical need; therefore, it had the benefit of PRIME, "EMA's platform for early and enhanced dialogue with developers of promising new medicines," the news release explains. As a result, the drug's application package was more robust and the drug was assessed in a more expeditious 150 days.

EMA news release. Published online January 31, 2020. Full text

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