Lily's orphan drug 'Jaypirca' receives conditional approval

The Central Pharmacist Review Committee decided on conditional approval requiring the submission of Phase 3 study results

Clinical trials showing therapeutic confirmation are expected to be reported by March 2027

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'Jaypirca,' a designated orphan drug for the treatment of Mantle Cell Lymphoma that recently received marketing authorization in South Korea, is expected to submit the Phase 3 trial results showing therapeutic confirmation by March 2027.

 

As it received conditional approval based on Phase ½ clinical trial results, the company must compare Jaypirca to existing Bruton tyrosine kinase (BTK) inhibitors and confirm clinical benefits.

 

According to the meeting notes of the Central Pharmacist Review Committee, disclosed by the Ministry of Food and Drug Safety (MFDS) on August 27th, the committee members exchanged opinions on conditional approval of Jaypirca, an orphan drug for treating Mantle Cell Lymphoma (MCL).

 

Jaypirca has received domestic approval for its efficacy and effects as a 'monotherapy for adult patients with relapsed or refractory MCL previously received at least two treatments.' Because this drug works differently in BTK-binding compared to existing treatments, Jaypirca has the advantage of treating MCL patients who are difficult to treat with existing treatments, such as 'Imbruvica (ibrutinib)' and 'Brukinsa (zanubrutinib).' However, similar to drugs of the same type, Jaypirca's Phase 2 clinical trial outcomes did not lead to improved survival in the Phase 3 trial.

 

The expert opinion suggested that the decision on the clinical effectiveness of Jaypirca cannot be made based on the current outcomes.

 

Jaypirca received marketing authorization based on Phase ½ clinical trial outcome as an orphan drug.

 

It has been suggested that additional clinical trials are needed to confirm clinical effectiveness for the consideration of approval.

 

A Central Pharmacist Review Committee member commented, "Although the global Phase 3 trial is being conducted to secure therapeutic confirmation, the trial's patient group differs from the target indication for approval.

 

Thus, it is difficult to use the outcomes as the basis of approval," and added, "Objective response rate is not sufficient to decide on the final clinical effectiveness.

 

Therefore, we need to grant conditional approval requesting the Phase 3 clinical trial showing therapeutic confirmation." Another member stated, "The drug meets the requirement for orphan drug designation.

 

However, additional documents are needed to evaluate the final clinical effectiveness and benefits," and added, "As a condition of approval, we must require Phase 3 clinical trial showing the final clinical effectiveness and clinical benefits over existing BTK treatments." However, some argue that the drug's marketing authorization is necessary because existing BTK inhibitors target MCL patients who do not have alternative treatment options.

 

A committed member said, "There are no existing treatment methods using BTK inhibitors in South Korea.

 

The submitted Phase 1/2 clinical trial results show an overall response rate (ORR) of 57.8% and a duration of response (DOR) close to one year, meeting the efficacy targets and showing no significant issues in safety evaluations." Some argue that Jaypirca's approval is necessary because CAR-T for blood cancer treatments is expensive and has not been approved as an MCL treatment.

 

A committee member said, "Previously, there were no treatment methods using BTK inhibitors in South Korea," and emphasized, "While there are no significant issues with safety evaluations, it is advisable to grant conditional approval based on the submission of Phase 3 data, as Phase 2 clinical trials alone cannot definitively establish the stability and efficacy of the treatment." Consequently, Jaypirca received conditional approval despite not having the Phase 3 document.

 

The approval has been made due to the patient's need for treatments, with the expectations that the company will submit the clinical outcomes showing therapeutic confirmation by March 2027."