Agios leukemia drug Tibsovo has been approved in the United States for its cold adaptation in Europe.

Recently, Agios said it had withdrawn a sales license application for the acute leukemia drug Tibsovo (ivosidenib) in the European market, mainly for the recurrence or refrhed acute myeloid leukemia (R/R AML) of the IDH1 (isocric acid dehydrogenase-1) mutation.
, the drug had been approved by the FDA for the allergy.
, European regulators do not agree with the FDA's approval decision.
reason, the European Medicines Agency's (EMA) Commission on Human Pharmaceutical Products (CHMP) found that the data from the company's one-arm, open Phase I clinical trials were not sufficient to justify the clinical benefits and risks of the drug.
the FDA approved the drug's U.S. listing in July 2018, based on the above-mentioned Phase I clinical trial data.
addition, the drug has been identified by the FDA as a breakthrough therapy for the treatment of recurring or refragsive bone marrow growth abnormal syndrome (MDS) that carries the IDH1 mutation.
SVB Leerink analyst Andrew Berens cut his forecast for Tibsovo revenue in Europe to $159 million from $237 million, saying the rejection of the application reduced the "fundamental value" of the first IDH1 inhibitor franchise.
, founder of Endpoints News, said the failure highlighted the risks involved in FDA Director Richard Pazdur's rapid completion of approvals.
, however, is not a complete loss of the European market, and for now, Tibsovo's remaining opportunities in Europe lie with patients who have not previously received treatment.
Agios is conducting two phase III trials of newly diagnosed AML patients, which link Tibsovo to new-based Vidaza, to treat patients who are not suitable for chemotherapy, and another Hovon150 trial, which tested the efficacy of Tybsovo's combination chemotherapy therapy.
said Agios plans to apply for market approval for the adaptation in the U.S. and the European Union if the trials yield positive results.
it's worth noting that in December, Idhifa, an IDH inhibitor of BMS, also withdrew its application for a new drug in the European market, which had previously been approved by the FDA to treat AML for IDH2 mutations, and that Idhifa failed to show in Phase 3 trials that drug therapy can extend a patient's life.
, Agios believes that patients with IDH1 mutations have a worse prognosis than those with IDH2 abnormalities, and that randomized trials of patients with relapsed or difficult-to-treat IDH1 are not feasible.
Tybsovo has also been approved in the U.S. market for single-drug therapies for new diagnostic AML patients who do not meet the requirements of chemotherapy.
Agios hopes to use the drug for previously treated IDH1 mutant bile tube cancer, a rare bile tube cancer that Tibsovo showed in Phase 3 trials can significantly reduce the risk of disease progression in patients, and subjects showed a trend toward improved survival.
addition to Tibsovo, another IDH inhibitor, vorasidenib, is also in the development phase 3 trial.
source: Agios' Tibsovo has an FDA green light in AML. Why did European regulators turn it back?