PharmaEssentia announced on the 21st that its Japanese subsidiary has completed a submission to change the dosage instructions for its new drug, Ropeginterferon alfa-2b (Ropeg), following market approval. The proposed change would adjust the current treatment regimen for adult patients with polycythemia vera (PV) from an initial dose of 100 micrograms, increasing by 50 micrograms every two weeks, to a new regimen starting at 250 micrograms and increasing to the target dose of 500 micrograms within four weeks.
PharmaEssentia stated that the high-dose Ropeg regimen would deliver faster therapeutic benefits to patients, helping to increase shipments of Ropeg in the Japanese market and drive sales growth.
Ropeg received approval for PV treatment in Japan in March 2023, specifically for patients who have insufficient response to or are unsuitable for existing treatment options. The current recommended regimen starts with 100 micrograms, with increments of 50 micrograms every two weeks, reaching a maximum dose of 500 micrograms. The proposed change would increase the starting dose to 250 micrograms, followed by increments to 350 micrograms after two weeks and 500 micrograms after another two weeks, with a maintenance dose administered every two weeks thereafter.
PharmaEssentia's CEO, Ko-Chung Lin, emphasized that clinical data has demonstrated Ropeg's good tolerability, and the high-dose Ropeg regimen effectively prevents PV disease progression. Achieving the target dose more rapidly would expedite the therapeutic goals of PV treatment. The application for a label change in Japan is a significant milestone for the company’s years of efforts to promote high-dose regimens and is key to expanding its presence in the Japanese market. It is expected to be approved within the year and will significantly support the growth of Ropeg shipments and market share in Japan.
Ko-Chung Lin further pointed out that the Phase III clinical trial (PROUD-PV) of Ropeg in Europe was unsuccessful due to slow dose escalation, preventing the treatment’s full effects from being realized in the first year. In contrast, the U.S. FDA approved Ropeg for primary thrombocythemia (ET) in 2019 based on a high-dose regimen (250-350-500 micrograms) in its global Phase III trials, with highly positive statistical results published in January, including a p-value of 0.0001. Clinical research data from Taiwan, China, and South Korea also confirmed that the high-dose regimen of Ropeg has good tolerability and can show therapeutic effects more quickly.
Since Ropeg’s official launch in Japan in June 2023, PharmaEssentia has started clinical trials for the high-dose regimen in Japan as planned. By the sixth month, the complete hematologic response (CHR) rate reached 57.1%, significantly higher than the 31.2% seen in the European PROUD-PV trial. Clinical trial data showed that the high-dose regimen of Ropeg not only effectively reduced blood cell counts in patients’ peripheral blood vessels but also reduced the need for phlebotomy treatments. More than 80% of patients significantly lowered the JAK2 V617F allele burden, a key mutation gene that triggers PV, without new safety concerns. No patients dropped out or stopped the treatment during the trial, confirming that the high-dose regimen exhibited excellent tolerability and clinical benefits in the Japanese PV patient population.
PharmaEssentia added that the relationship between doctors and patients in Japan is very close, and the discontinuation rate for medications is very low. In addition, the company’s Japanese team is actively strengthening its market presence, including providing patient support services, identifying potential PV patients, and promoting testing for JAK2 mutations. These efforts will help ensure long-term Ropeg usage by patients, with an increased likelihood of using the high-dose regimen. Starting in June 2024, Japan's National Health Insurance will allow patients to self-administer Ropeg and extend the prescription period to up to 90 days, reducing the need for frequent hospital visits and lowering the medical cost burden. This will significantly improve the convenience and willingness of patients to continue treatment. Ko-Chung Lin added that this submission for the high-dose regimen is just the first step, and the company will actively promote the use and approval of this regimen globally. PharmaEssentia is confident that expanding the high-dose Ropeg regimen will bring significant benefits to a broader population of PV patients and expects the Japanese market to continue its accelerated growth.
Resource: 藥華藥日本申請Ropeg高劑量方案 可望提升出貨量
