Supply of Acute Hepatic Porphyria Therapy Givlaari Unlikely Affected by Pandemic

Supply of Acute Hepatic Porphyria Therapy Givlaari Unlikely Affected by Pandemic
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Alnylam Pharmaceuticals is not expecting the production and supply of Givlaari (givosiran), a treatment for acute hepatic porphyria (AHP), to be affected by the COVID-19 pandemic, the company said.

Besides Givlaari, Alnylam also markets Onpattro (patisiran), which is approved in the U.S. for treating familial amyloid polyneuropathy (FAP). 

“Currently we have sufficient inventory of drug and raw materials to meet our projected demand plus appropriate safety stock and don’t at this time anticipate any significant impact from COVID-19 on the manufacture of our commercial products or product candidates in clinical studies,” Alnylam said on its website.

The company is offering region-specific resources to patients, caregivers, and healthcare providers who need support related to their medications. In the United States, the support program Alnylam Assist can be reached online or by phone at 833–256–2748.

People outside of the U.S. can email [email protected] or call the following phone numbers, depending on where they live: Europe, 31–20–369–7861; Canada, 877–356–9526; Japan, 03–4520–8966; and Brazil, 0800–047–4597.

Alnylam also announced that it is taking steps to ensure the safety of its workers and minimize COVID-19 spread. This has included work-from-home orders for everyone who can, cutting non-essential travel, and proving assistance programs to employees.

In addition, the company is making efforts to support its local community, including monetary donations to the Boston Resiliency Fund and the Life Sciences Cares COVID-19 Response Fund, as well as donations of supplies, including personal protective equipment (PPE), to Boston-area hospitals.

Alnylam also is continuing a collaboration with Vir Biotechnologyannounced in early March, to develop potential treatments for COVID-19. Alnylam’s technology (including Givlaari and Onpattro) is based around RNA interference (RNAi), an approach that targets a specific gene to stop it from being translated into protein — in essence, “turning off” the gene.

Givlaari works by degrading the messenger RNA generated from the ALAS1 gene to prevent further production of the ALAS1 enzyme. This is intended to reduce the toxic levels of compounds called aminolevulinic acid and porphobilinogen inside cells.

In the context of COVID-19, the goal is to find RNAi candidates that can “turn off” critical genes for SARS-CoV-2 (the virus that causes COVID-19).

Early work done by Alnylam has identified more than 350 possible candidates, which Vir is evaluating for its anti-viral activity. A similar collaboration between these companies resulted in VIR-2218, a potential treatment for hepatitis B, that is currently being tested in a Phase 1/2 clinical trial (NCT03672188).

“During a moment like this, it’s incumbent upon all of us in the global life sciences community to harness the full potential of our technologies, pipelines, people and products to assess how we can address public health needs,” said John Maraganore, the CEO of Alnylam.

“Based on our understanding of SARS-CoV-2, the features of Alnylam’s RNAi therapeutics technology, and our existing work in novel forms of drug delivery, we believe that RNAi therapeutics represent a promising approach for targeting coronaviruses, like SARS-CoV-2,” Maraganore said.

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.
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Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.
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