Shares of AstraZeneca plc (ADR) (NYSE:AZN) ended Thursday session in red amid volatile trading. The shares closed down -0.78 points or -2.29% at $33.05 with 6.97 million shares getting traded. Post opening the session at $33.44, the shares hit an intraday low of $32.77 and an intraday high of $33.47 and the price vacillated in this range throughout the day. The company has a market cap of $82.95 billion and the numbers of outstanding shares have been calculated to be 1.26 billion shares.
AstraZeneca plc (ADR) (AZN) on September 29, 2016 announced that the US Food and Drug Administration (FDA) has approved a blood-based companion diagnostic for TAGRISSO® (osimertinib). The companion diagnostic for TAGRISSO is the only FDA-approved and clinically validated companion diagnostic test that uses either tissue or a blood sample to confirm the presence of a T790M mutation in patients with metastatic epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC), who have progressed on or after an EGFR tyrosine kinase inhibitor (TKI) medicine.
The approval provides a new, non-invasive option to identify patients with metastatic EGFR T790M mutation-positive NSCLC, ensuring that those patients who may not be suitable for biopsy procedures have an opportunity to be tested. Blood-based testing for the presence of the mutation is recommended only when a tumor biopsy cannot be obtained. Patients who test negative for the T790M mutation with the blood-based test, and their physicians, should re-evaluate the feasibility of tissue-based testing to confirm the presence of the EGFR T790M mutation.
The companion diagnostic, cobas® EGFR Mutation Test v2, was developed by Roche Molecular Systems. The test enables identification of patients who have the T790M mutation at disease progression, and is initially available through Baystate Health, Carolinas HealthCare System, Laboratory Corporation of America® Holdings (LabCorp®), and PhenoPath.
“Blood-based testing has the potential to rapidly identify patients eligible for targeted therapy, who may not be eligible for biopsy. Availability of this blood-based test may help aid treatment decisions,” said Balazs Halmos, MD, Montefiore Medical Park, Albert Einstein College of Medicine.
“The availability of an FDA-approved, blood-based companion diagnostic is a tremendous step forward for patients with lung cancer in need of a high-quality test that provides results with a rapid turnaround time. This development offers an important option for the identification of the T790M mutation in patients with metastatic EGFR mutation-positive NSCLC who have progressed on an EGFR TKI medicine, for whom a tissue biopsy may not be feasible,” said Andrew Coop, Vice President, US Medical Affairs, Oncology, AstraZeneca. “Delivering targeted therapies, such as TAGRISSO, to the right patients at the right time demonstrates our commitment to testing and quality companion diagnostics.”
Shares of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) ended Thursday session in red amid volatile trading. The shares closed down -3.25 points or -3.38% at $92.78 with 1.05 million shares getting traded. Post opening the session at $96.02, the shares hit an intraday low of $92.43 and an intraday high of $96.17 and the price vacillated in this range throughout the day. The company has a market cap of $15.68billion and the numbers of outstanding shares have been calculated to be 170.78 million shares.
BioMarin Pharmaceutical Inc. (BMRN) on Sept. 15, 2016 announced that the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for Brineura™ (cerliponase alfa) to treat children with CLN2 disease, a form of Batten disease. Validation of the MAA confirms that the submission is accepted and starts the formal review process by the EMA’s Committee for Human Medicinal Products (CHMP).
Earlier this year, the CHMP accepted BioMarin’s request for accelerated assessment of the MAA on the grounds that Brineura is of major public health interest with the potential to have a major impact on medical practice for CLN2 patients in Europe. Accelerated assessment has the potential to shorten the EMA’s review procedure. However, applications which are initially granted accelerated assessment frequently revert to standard assessment timelines. Assuming a positive opinion from the CHMP and standard assessment timing, a decision from the European Commission is anticipated by the third quarter of 2017. The EMA previously granted Brineura Orphan Drug Designation.
“CLN2 disease is a rapidly progressing, fatal neurodegenerative disease with no approved treatments. Based on the positive results of the pivotal study, we are working to bring this meaningful therapeutic option to patients and families in Europe as soon as possible,” said Hank Fuchs, MD, Chief Medical Officer of BioMarin. “We greatly appreciate the CLN2 community’s ongoing support and look forward to continuing to work with regulatory authorities in the coming months.”