Novartis Rydapt wins EU approval for AML, other diseases

ZURICH: Swiss drugmaker Novartis on Wednesday won European Union approval for Rydapt to be used against a mutated form of acute myeloid leukemia (AML) and other rare diseases, adding to U.S. approvals the medicine secured in April.

Rydapt was cleared to be used along with chemotherapy to treat adults newly diagnosed with AML who have a specific genetic mutation called FLT3, Novartis said.

The drug was also approved for three types of advanced systemic mastocytosis (ASM), a rare disease where mast cells accumulate in the skin, bone marrow, and some internal organs.

AML is cancer that originates in the bone marrow and progresses rapidly, resulting in an abnormal increase in white blood cells. Rydapt, among Novartis's stable of potential $1 billion-per-year medicines, is targeted at those with a tough-to-treat form of the disease whose options have been limited.

"For patients with FLT3-mutated AML, there have been no meaningful advancements in more than 25 years and with Rydapt they now have a targeted medicine that could significantly extend their lives," said Bruno Strigini, head of Novartis's cancer drugs business.

Using Rydapt for FLT3-mutated AML patients resulted in a 23 percent reduction in the risk of death, Novartis has said.

After its U.S. approval, analysts expect the drug will also find immediate uptake in Europe.

"We expect high penetration rates as well as high prices because the disease is very serious and there is no competition," said Michael Nawrath, a Zuercher Kantonalbank analyst, in a note to investors.

Nawrath forecasts $1.1 billion in annual sales in 2025.

Novartis did not immediately give European pricing details.

In the United States, Rydapt's list price for AML patients is $7,495 for a 14-day treatment and $14,990 for a 28-day treatment. In a late-stage trial on AML patients, the median duration of therapy was 42 days, implying a list price of $22,485.

For ASM patients, the U.S. list price is $32,121 for a 30-day treatment duration, with the median duration of therapy in Novartis's clinical trials of 11.4 months.

Novartis is continuing to study Rydapt in a late-stage trial in patients with AML without an FLT3 mutation, which could result in its expanded use.