Alnylam's Onpattro sales look 'disappointing' in early days of RNAi launch: analyst

John Maraganore
Alnylam CEO John Maraganore (Alnylam)

It may be early days for Alnylam’s pioneering RNAi therapy Onpattro, but at least so far, sales look “disappointing,” according to one analyst.

That was the word from Jefferies’ Maury Raycroft after the New York biotech reported its third-quarter numbers, showing that between its August approval—as a treatment for peripheral nerve disease caused by the abnormal protein condition hereditary ATTR amyloidosis—and the end of the quarter, Onpattro racked up $460,000 in revenue.

Alnylam itself was much more upbeat, highlighting 125 patient start forms, or “requests that come to our Alnylam Assist patient hub to guide fulfillment of an Onpattro prescription by a physician,” company president Barry Greene explained on Alnylam’s third-quarter conference call. Sixty percent of those came from patients who had participated in the drugmaker’s early access program, he said.

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With 200 patients around the world in the program, that leaves about 125 of them who have yet to convert, Raycroft noted, adding that about 25 of those patients were U.S.-based. Those figures fueled his prediction that 185 patients would be on the drug by the end of the year.

RELATED: Alnylam vows value-based pricing, financial help with $450K Onpattro launch

Greene, though, reminded investors that at this point in the rollout, “it will take a few months to get patients started on commercial drug after receiving the start form. We do expect this timeline to decrease significantly as our launch progresses.”

While Raycroft warned investors not to “read into” the launch performance “too much, given the early stage,” he did add that after Alnylam converts the rest of its U.S. early access patients, the company’s “internal commercial efforts from the past year will have to translate.”

RELATED: Relative power: Alnylam taps family to highlight hereditary amyloidosis in new campaign

But meanwhile, the company isn’t letting up. Its medical affairs team is continuing to work “to raise disease awareness and improve diagnosis,” Greene said, and it’s fielding a third-party genetic screening initiative to help with diagnosis, too. Alnylam is hoping those efforts will help it diagnose more of the 20,000 to 30,000 hATTR amyloidosis patients with polyneuropathy that it estimates are out there worldwide; currently only about 5,000 are diagnosed, with just 3,000 in the U.S., according to the company’s predictions.  

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