A roundup of the latest market developments from the pipelines of the pharmaceutical majors, featuring news from Pfizer and Astellas Pharma.

Editor’s Note: This article was updated on a continuous basis for news announced from Wednesday, May 23, 2018 to Tuesday, May 29, 2018.

FDA Grants Breakthrough Therapy Designation for Pfizer Heart Failure Drug
The US Food and Drug Administration (FDA) has granted breakthrough therapy designation to Pfizer’s tafamidis for treating patients with transthyretin cardiomyopathy, a rare, fatal, and underdiagnosed condition associated with progressive heart failure.

Tafamidis is an investigational treatment for transthyretin cardiomyopathy and is not yet approved for this indication. In 2012, tafamidis was granted orphan-drug designation for transthyretin cardiomyopathy in both the European Union and US. In May 2017, the FDA granted fast-track designation to tafamidis for transthyretin cardiomyopathy. In March 2018, the Ministry of Labor Health and Welfare in Japan granted the drug Sakigake designation, a policy for early introduction of medicines and medical devices that are initially developed in Japan, for this indication.

Source: Pfizer

FDA Grants Breakthrough Therapy Designation for New Uses of Pfizer’s Cancer Drug Xalkori
The US Food and Drug Administration (FDA) has granted breakthrough therapy designation for two new uses for Pfizer’s Xalkori (crizotinib) for treating particular patients with metastatic non-small cell lung cancer (NSCLC).

In one indication, the FDA has approved the Xalkori for treating patients with mesenchymal-to-epithelial transition (MET) exon 14 alterations with disease progression on or after platinum-based chemotherapy. MET is a transmembrane tyrosine receptor kinase, which is expressed in several types of cells, according to information from Pfizer. In the second indication, the FDA granted breakthrough therapy designation for Xalkori for treating patients with relapsed or refractory systemic anaplastic large cell lymphoma that is anaplastic lymphoma kinase (ALK)-positive.

Xalkori is a tyrosine kinase inhibitor indicated for treating patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are anaplastic lymphoma kinase (ALK) or c-ros oncogene 1 (ROS1-positive) as detected by an FDA-approved test. The drug has received approval for patients with ALK-positive NSCLC in more than 90 countries including Australia, Canada, China, Japan, South Korea, and the European Union. Xalkori is also approved for ROS1-positive NSCLC in more than 60 countries.

Source: Pfizer

FDA Grants Priority Review to Astellas’ NDA for Leukemia Drug
The US Food and Drug Administration (FDA) has granted priority review to Astellas Pharma’s new drug application (NDA) for gilteritinib for treating adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a tyrosine kinase 3 mutation mutation, as detected by an FDA-approved test.

The FDA grants priority-review designation to applications for drugs that, if approved, may offer improvements in the safety and effectiveness of the treatment of serious conditions when compared to standard applications. Under priority review, the FDA aims to take action on an application within six months of NDA filing, as compared to ten months under standard review.

Previously, gilteritinib was granted both orphan-drug designation and fast-track designation by the FDA. Gilteritinib also received orphan-drug designation from the European Commission and orphan-drug designation from the Japan Ministry of Health, Labor and Welfare (MHLW). The MHLW also granted Sakigake designation, a policy for early introduction of medicines and medical devices that are initially developed in Japan, to gilteritinib for relapsed/refractory AML.

Source: Astellas Pharma