Jaypirca

Jaypirca (pirtobrutinib) is a drug that received a positive opinion for conditional marketing authorisation on April 26, 2023. It was designated as an orphan medicinal product on June 21, 2021, but this designation was removed from the Union Register on October 30, 2023. The marketing authorisation application was submitted on May 25, 2022, following an agreement on eligibility to the centralised procedure on February 25, 2021. An accelerated assessment request was declined.

In a Phase 2 study, Jaypirca demonstrated an Overall Response Rate (ORR) by Independent Review Committee (IRC) of 51.8% and a Duration of Response (DOR) by IRC of 7.46 months. The Progression-Free Survival (PFS) by IRC was 5.55 months, and Overall Survival (OS) was 14.75 months. In a broader analysis, the ORR by IRC was 57.8%, with a DOR by IRC of 21.59 months and a PFS by IRC of 7.36 months. Preclinical studies showed tumour growth inhibition in REC 1 MCL xenograft models, with 80% at 10 mg/kg BID and 84% at 30 mg/kg BID, and tumour regression of -11% at 50 mg/kg BID.

Regarding safety, the rate of any Serious Adverse Event (SAE) was 33.5% (MSAS) and 38.6% (CSAS). Related SAEs occurred in 4.9% (MSAS) and 8.4% (CSAS) of patients. Pneumonia was reported in 4.7% of patients. The No Observed Adverse Effect Level (NOAEL) for pirtobrutinib in male rats was 500 mg/kg BID in 14-day and 28-day studies, and 50 mg/kg BID in a 3-month study. For female rats, the NOAEL was 75 mg/kg BID in a 14-day study and 175 mg/kg BID in a 28-day study.