Alnylam’s first-in-class drug approved, manufacturing no ‘trivial undertaking’

By Ben Hargreaves contact

- Last updated on GMT

(Image: Getty/Ktsimage)
(Image: Getty/Ktsimage)

Related tags: Alnylam, RNAi, Onpattro

Alnylam received US FDA approval for its first-in-class RNAi treatment, Onpattro, but a spokesperson confirmed manufacturing for the treatment is a complicated affair.

Alnylam’s long road to bringing its first RNAi therapy to market has concluded with the successful approval by the US Food and Drug Administration of Onpattro (patisiran), after working in the RNAi (RNA interference) space since 2002.

The Boston-based biotech’s treatment has been approved for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

This type of RNAi treatment functions by ‘silencing’ certain genes, a discovery that won the 2006 Nobel Prize for Physiology and Medicine. In this case, Onpattro prevents the production of disease-causing proteins for hAATR.

Approximately 3,000 people are living with the condition in the US, where the product is initially approved, and the company has priced the treatment at $450,000 per patient, per year.

A spokesperson for the company explained how the biotech arrived at this pricing: “When determining the price of Onpattro, many key factors were considered, including the urgent unmet need for patients living with this condition, Onpattro’s ability to reverse neuropathy impairment, the rare nature of hATTR amyloidosis and the years of research and development we’ve invested into the treatment.”

Revolutionary treatment, fundamental manufacturing requirements

The manufacturing process for the treatment is not a simple path, as Alnylam’s spokesperson explained to us: Onpattro is delivered in a single vial containing its active pharmaceutical ingredient (API) encapsulated within Lipid Nano Particles (LNP). Onpattro API (small interfering Ribonucleic Acid—siRNA) is manufactured using traditional solid-phase chemical synthesis, common in the manufacturing of oligonucleotides and peptides.”

The spokesperson continued, “To encapsulate siRNA API into LNPs, a solution containing siRNA is mixed under tightly controlled conditions, with the proprietary solution of lipids to yield LNPs. Additional manufacturing steps are required to ensure purity and the correct dosage strength prior to the product filled into a vial.”

The spokesperson highlighted that this last step is of particular difficulty, despite being common to the manufacture of most treatments. Ensuring purity, the spokesperson noted, is not a “trivial undertaking”​ and requires a “robust set of manufacturing control”​.

We previously reported that Agilent Technologies will be responsible for the manufacture of the commercial treatment​, which is currently approved for sale only in the US. However, the European Medicine’s Agency has recommended the treatment for regulatory approval by the European Commission in July​.

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