Ionis’ Tegsedi launches into head-to-head with Alnylam, but Pfizer’s waiting in the wings

Akcea's hATTR drug Tegsedi comes in a prefilled syringe, which gives patients the flexibility to self-administer at a time that works for them. (Akcea Therapeutics)

It is game on between Ionis and Alnylam in hereditary transthyretin-mediated amyloidosis (hATTR), and to make the competition more interesting, Pfizer could soon join the party.

Not only has Ionis’ affiliate Akcea just won an FDA nod for Tegsedi in polyneuropathy ATTR—for which Onpattro was approved in August—but it’s pricing the RNA drug at the exact same price as the Alnylam therapy. “We’re ready to go,” Akcea CEO Paula Soteropoulos said on a Friday conference call, as the company rolled out a detailed commercial plan.

Tegsedi comes in a prefilled syringe, which patients can administer subcutaneously by themselves. At the recommended dosing of once weekly, its annual list price will be $450,000. In contrast, at the same annual list price, Alnylam’s Onpattro needs to be given intravenously by a trained medical professional once every three weeks.

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Obviously, Tegsedi is more convenient for patients to take, and it does save on administration costs and drug wastage. But that advantage is compromised by a boxed warning slapped on its label.

The drug carries warnings for thrombocytopenia, or low platelet counts, and glomerulonephritis, which injures the kidney, said Akcea President Sarah Boyce on the call. The agency is asking doctors to take platelet counts every week and test kidney function every two weeks. If either of those numbers falls below its accepted threshold, the patient will be pulled off the treatment until the numbers return to normal.

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

Boyce noted that the FDA-required frequency is more stringent than what the company had proposed and what the European Medicines Agency and Health Canada have agreed to along with their recent approvals. But the company hopes to generate more data to support future adjustment to this schedule.

Nevertheless, in the eyes of the Institute for Clinical and Economic Review the $450,000 tag for both drugs is way too high to be considered cost-effective. In a recent report (PDF), the drug cost watchdog suggests both of them will need to take more than 90% off their list price to reach the minimum threshold of $150,000 per quality-adjusted life-year (QALY) gained. And it suggests insurers and other payers develop prior authorization criteria “to ensure prudent use of these treatments.”

Meanwhile, just as Alnylam said it would work out value-based pricing agreements with insurers, Akcea is considering “innovative access strategies such as value- and outcomes-based access arrangements in addition to other contracting strategies and risk-based approaches,” said Soteropoulos.

Akcea has partnered with Express Scripts’ specialty pharmacy Accredo to distribute Tegsedi “because of its experience supporting the unique needs of rare disease communities and its proven track record for simplifying access to therapy,” said the company. Accredo’s team of clinicians, pharmacists and more than 600 field-based nurses will also help Akcea’s own patient support team.

RELATED: Pfizer's tafamidis score still leaves 'door open' for Alnylam rival: analysts

As Ionis and Alnylam go head-to-head in hATTR polyneuropathy, their fight will likely be complicated by a third player—a Big Pharma—that has also thrown its cap in the ring.

Pfizer just in August announced positive data for its ATTR candidate tafamidis in ATTR cardiomyopathy (ATTR-CM). Although neuro and heart are two different indications within ATTR, these products will likely tap into each other’s territory. In fact, the Alnylam drug is already heading for cardiomyopathy, and Pfizer could eventually go the other way around, too, Evercore ISI and Credit Suisse analysts noted in separate memos to investors at the time.

Pfizer’s cardiomyopathy exceeded physicians’ and investors’ expectations, but as the analysts mentioned, the wrinkle in its case is that tafamidis’ high-dose version didn’t yield stronger results, which means it may not win over Onpattro in neuropathy. What’s more, it also didn’t show significant benefit in those with hereditary ATTR.