Data from Pharmawand - Curated by EPG Health - Date added 22 November 2020

Alnylam Pharmaceuticals, Inc. announces a new and enhanced framework for value-based agreements (VBAs) designed to help patients with acute hepatic porphyria (AHP) – an ultra-rare orphan disease – gain access to Givlaari (givosiran) injection for subcutaneous use, a first-of-its-kind RNAi therapeutic for the treatment of AHP. Approved by the FDA, Givlaari is indicated for the treatment of adults with AHP.

Alnylam is in active discussions with leading payers about VBAs for Givlaari and plans to incorporate this new ultra-rare disease framework into these discussions and negotiations. Alnylam has reached an agreement in principle with Harvard Pilgrim covering Givlaari. Under this innovative framework for VBAs, participating government and commercial payers will pay the full value for Givlaari only when it delivers patient outcomes in the real-world setting similar to results demonstrated in clinical trials.

An additional and newly designed Prevalence-Based Adjustment (PBA) feature will trigger rebates to participating payers if the number of diagnosed patients they cover exceeds current epidemiologic estimates for AHP. There are often uncertainties in diagnosis rates and disease prevalence estimates in ultra-rare disease diseases, making it challenging for payers to predict the number of patients who will be covered within their plans. This innovative approach offers greater certainty to payers that their overall financial risk will be adjusted if a substantially larger number of patients than currently estimated are identified, diagnosed, and treated with Givlaari.

Currently, the population of AHP patients with diagnosed, active disease in the U.S. and Europe is estimated to be approximately 3,000. Due to the broad, non-specific range of symptoms and comorbidities experienced by AHP patients, the time to diagnosis can be as long as 15 years.

In August, Alnylam announced a U.S. gastrointestinal (GI) disease education and promotional agreement for Givlaari with Ironwood Pharmaceuticals, Inc., a GI healthcare company. Under the agreement, Alnylam will leverage Ironwood’s leading capabilities in GI to help raise awareness of AHP among gastroenterologists and other healthcare practitioners in the U.S. Ironwood will also participate in Givlaari promotional efforts, augmenting Alnylam’s broader commercialization activities.


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