- The Food and Drug Administration has for the first time approved a therapy for relapsed or refractory acute myeloid leukemia (AML) patients who have a relatively uncommon genetic alteration called isocitrate dehydrogenase-1 (IDH1) mutation.
- Research indicates the mutation occurs in about 6% to 10% of AML patients and may negatively affect disease-free survival. Agios Pharmaceuticals' Tibsovo inhibits the IDH1 enzyme and showed a combined complete remission and complete remission with partial hematologic improvement rate of 32.8% in the Phase 1 trial that led to its Friday approval. The drug comes to market with a black box warning for differentiation syndrome, which can cause unexplained fever, organ problems and other symptoms that could be fatal.
- Agios is looking at filing Tibsovo with European regulators in the fourth quarter, as well as evaluating the drug in more clinical trials, including one that pairs it with the biotech's other marketed treatment, Idhifa, in frontline AML patients with IDH1 or IDH2 mutations.
Tibsovo (ivosidenib) gives Agios a chance to prove its marketing capabilities.
Though the biotech secured its first FDA approval last August with Idhifa (enasidenib), its partner Celgene holds worldwide development and commercialization rights to the drug. Agios does get royalty payments from Idhifa sales and is eligible for up to $95 million in milestone payments tied to European regulatory and commercial achievements — yet it still hasn't gotten any revenue directly from product sales.
Agios is therefore planning a rapid rollout of wholly-owned Tibsovo. Company leadership noted on a Friday call with investors that the drug would be available through a limited specialty pharmacy network within the next two days. A 30-day supply of the drug is priced at $26,115.
Executives also explained that the biotech's U.S. sales force of about 32 should benefit from its experience promoting Idhifa, which was greenlighted for relapsed or refractory AML patients with IDH2 mutations.
"This team is now going to be promoting two products: Tibsovo as well as Idhifa," Steve Hoerter, Agios' chief commercial officer, said on the call. "This is the same customer base, often will be the exact same sales call that we would expect both products to be discussed. And so that team now, upon finalization of training, will begin promoting Tibsovo very early next week."
And while U.S. commercialization efforts are just getting underway, Agios indicated the possibility of a European rollout in the near future is also top of mind.
"Obviously access would be most important to us. And depending upon the timeline for potential approval outside the United States, that would dictate whether or not we will seek a partner or whether we would potentially build on our own, and it's just a little bit too early to give you a final answer," Agios CEO David Schenkein said on the call, referring to establishing a Tibsovo sales force in Europe.