Pfizer’s lorlatinib got compassionate use the most in 2020

Pfizer’s lung cancer drug lorlatinib (brand name in U.S.: Lorbrena) obtained the regulator’s nod for therapeutic purposes the most among experimental treatments in Korea in 2020, government data showed.

The Ministry of Food and Drug Safety’s data, analyzed by Korea Biomedical Review, demonstrated that the regulator granted the use of investigational drugs for 485 cases for therapeutic purposes last year. Among them, lorlatinib use accounted for 187 cases, or 39 percent.

The ministry allows the use of an investigational treatment for therapeutic purposes to give treatment opportunities to patients with serious or life-threatening diseases or those with no other treatment options.

Pfizer’s investigational lung cancer drug lorlatinib received approval for therapeutic purposes the most in Korea in 2020.

The U.S. and Europe also operate similar systems such as the Expanded Access Program and Compassionate Use, respectively.

Lorlatinib is an investigational drug for non-small cell lung cancer (NSCLC) being developed by Pfizer as the successor to crizotinib (brand name: Xalcori).

Lorlatinib won the FDA nod as the second-line treatment for anaplastic lymphoma kinase (ALK)-positive NSCLC in the U.S.

The agent is showing efficacy in the first-line therapy, too.

In September last year, researchers announced at the European Society for Medical Oncology’s annual meeting that lorlatinib extended progression-free survival significantly, compared to crizotinib, in the first-line treatment for ALK-positive NSCLC patients.

The Korean government designated lorlatinib as an orphan drug for ALK-positive NSCLC in March. However, the drug has yet to win approval from the ministry.

The government approved the therapeutic use of lorlatinib because patients who failed in ALK-positive NSCLC therapy had no other treatment option other than chemotherapy, which is difficult for patients to endure.

The ministry also allowed Pfizer’s talazoparib, a poly ADP ribose polymerase (PARP) inhibitor for breast cancer treatment, to be used for therapeutic purposes in 36 cases.

The regulator granted the use of talazoparib for therapeutic purposes to treat adult patients with locally advanced or metastatic breast cancer with gBRCA mutation and human epithelial growth factor receptor 2 (HER2) negative, who had previously received chemotherapy, even after July last year when the MFDS gave the license.

A Covid-19 plasma therapy candidate GC5131 received approval for therapeutic use in 28 cases in 2020, followed by NSCLC drug pralsetinib (brand name: Gavreto) in 14 cases, myelofibrosis treatment Ruxolitinib (brand name: Jakavi) in 14 cases, and acute myeloid leukemia drug Gilteritinib (brand name: Xospata) in 14 cases.