FDA Assigns Sacituzumab Govitecan Breakthrough Designation for TNBC

“The FDA has granted sacituzumab govitecan (IMMU-132) breakthrough therapy designation for the treatment of patients with triple-negative breast cancer (TNBC) following at least 2 treatments for metastatic disease, according to Immunomedics, the manufacturer of the investigational antibody-drug conjugate.

“The designation, which will expedite the development and review of sacituzumab govitecan in TNBC, is based on a phase II trial in which the therapy induced a response rate of 31% in heavily pretreated patients with metastatic TNBC.

“ ‘We believe breakthrough therapy designation for IMMU-132 further validates this potential therapeutic for patients with TNBC, and we are delighted to receive this important recognition,’ Cynthia L. Sullivan, president and chief executive officer, of Immunomedics, said in a statement. ‘We continue to assess partnering opportunities while completing the scale-up manufacturing and regulatory activities for an international, randomized, controlled, registration trial in TNBC, based on the special protocol assessment agreement that was already granted by the FDA,’ she added.”


FDA Grants Olaparib Breakthrough Designation in mCRPC

“Olaparib (Lynparza) has received an FDA breakthrough therapy designation as a treatment for patients with BRCA1/2 or ATM-mutated metastatic castration-resistant prostate cancer (mCRPC) in those who have received a prior taxane-based chemotherapy and at least either hormonal agent enzalutamide (Xtandi) or abiraterone acetate (Zytiga).

“The designation, which will accelerate the development and review of the first-in-class oral PARP inhibitor, is based on data from the phase II TOPARP-A trial that demonstrated that olaparib monotherapy had an overall response rate (ORR) of nearly 90% in a biomarker-defined subgroup of patients who had DNA-repair defects.


Lilly Receives FDA Breakthrough Therapy Designation for Abemaciclib – a CDK 4 and 6 Inhibitor – in Advanced Breast Cancer

“Eli Lilly and Company (NYSE: LLY) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to abemaciclib, a cyclin-dependent kinase (CDK) 4 and 6 inhibitor, for patients with refractory hormone-receptor-positive (HR+) advanced or metastatic breast cancer. This designation is based on data from the breast cancer cohort expansion of the company’s Phase I trial, JPBA, which studied the efficacy and safety of abemaciclib in women with advanced or metastatic breast cancer. Patients in this cohort had received a median of seven prior systemic treatments. These data were presented at the San Antonio Breast Cancer Symposium in 2014.”


FDA Grants Nivolumab Priority Review in Nonsquamous NSCLC

“Nivolumab (Opdivo) has received an FDA priority review designation for patients with previously treated nonsquamous non­–small cell lung cancer (NSCLC), according to the developer of the PD-1 inhibitor, Bristol-Myers Squibb (BMS). Under the expedited process, the FDA’s decision deadline is January 2, 2016.

“The FDA simultaneously granted nivolumab a breakthrough therapy designation in this setting. The priority and breakthrough designations are based on data from the phase III CheckMate-057 trial, in which second-line nivolumab reduced the risk of death by 27% versus docetaxel in patients with nonsquamous NSCLC, including a 60% risk reduction among patients with the highest levels of PD-L1 expression.

“Nivolumab was previously approved in March 2015 for patients with squamous cell NSCLC who have progressed on or after platinum-based chemotherapy.”


FDA Grants Breakthrough Therapy Designation to Azedra for Pheochromocytoma, Paraganglioma

“The FDA granted breakthrough therapy designation to Ultratrace iobenguane I-131 for the treatment of patients with iobenguane-avid metastatic or recurrent pheochromocytoma and paraganglioma, according to a press release from the drug’s manufacturer.

“Ultratrace iobenguane I-131 (Azedra, Progenics Pharma), a late-state radiotherapeutic drug candidate, is being evaluated in a phase 2b trial of patients with malignant pheochromocytoma or paraganglioma. Pheochromocytomas — rare tumors usually found within one or both adrenal glands — are referred to as paragangliomas when they arise in other areas of sympathetic nerve cells.

“Ultratrace iobenguane I-131 — a very high specific activity form of iobenguane I-131 — is produced using Progenics’ Ultratrace platform, a technology designed to prevent unlabeled iobenguane from being carried through the manufacturing process to the final formulation, according to information listed on Progenics’ website. Ultratrace iobenguane I-131 is designed for use as an imaging agent in order to determine a therapeutic dose that is safe for normal organs, as well as for therapy.”


Xalkori Granted a Breakthrough Designation for ROS Positive NSCLC

“Pfizer Inc. announced today that XALKORI® (crizotinib) received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the potential treatment of patients with ROS1-positive non-small cell lung cancer (NSCLC). Occurring in approximately one percent of NSCLC cases1, ROS1-positive NSCLC represents a particular molecular subgroup of NSCLC.2 XALKORI currently is approved in the U.S. for the treatment of patients with metastatic NSCLC whose tumors are anaplastic lymphoma kinase (ALK)-positive as detected by an FDA-approved test.

“Enacted as part of the 2012 FDA Safety and Innovation Act (FDASIA), Breakthrough Therapy designation is intended to expedite the development and review of a potential new medicine if it is “intended to treat a serious or life-threatening disease and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies.”3The Breakthrough Therapy designation is distinct from the FDA’s other mechanisms to expedite drug development and review.4

“ ‘We are excited that the FDA has granted Breakthrough Therapy designation for XALKORI as a potential treatment for patients with ROS1-positive NSCLC,’ said Dr. Mace Rothenberg, senior vice president of Clinical Development and Medical Affairs and chief medical officer for Pfizer Oncology. ‘XALKORI pioneered precision medicine for ALK-positive metastatic NSCLC, and ROS1 represents a second molecular subgroup of NSCLC in which XALKORI has demonstrated a level of anti-tumor activity that can potentially make a real difference for patients.’ ”


Immune System-Boosting Drug Gets a Second FDA Breakthrough Designation, This Time for Lung Cancer

The gist: The U.S. Food and Drug Administration (FDA) has granted “breakthrough therapy designation” to the drug MPDL3280A for treating certain lung cancer patients. This designation means that review and approval will be accelerated so that MPDL3280A can more quickly reach patients in the U.S., outside of clinical trials. MPDL3280A is an immunotherapy drug, meaning that it boosts a patient’s own immune system to fight cancer. The breakthrough therapy designation specifically applies to patients with advanced non-small cell lung cancer (NSCLC) whose disease worsened despite treatment with platinum-based chemotherapy (e.g. carboplatin).

“Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that it has received a second Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for its investigational cancer immunotherapy MPDL3280A (anti-PDL1). The designation was granted for the treatment of people with PD-L1 (Programmed Death-Ligand 1) positive non-small cell lung cancer (NSCLC) whose disease has progressed during or after platinum-based chemotherapy (and an appropriate targeted therapy for those with an EGFR mutation-positive or ALK-positive tumor).

” ‘Lung cancer is the leading cause of cancer death globally, and we are pleased the FDA has granted breakthrough designation for MPDL3280A in non-small cell lung cancer,’ said Sandra Horning, M.D., chief medical officer and head of Global Product Development. ‘We are committed to personalized healthcare, developing medicines like MPDL3280A with companion tests that may help us identify those who may be appropriate candidates for our medicines.’ “


Merck Receives FDA Breakthrough Therapy Designation for KEYTRUDA® (pembrolizumab) in Advanced Non-Small Cell Lung Cancer

The gist: The U.S. Food and Drug Administration (FDA) has granted “breakthrough therapy designation” to the drug Keytruda (aka pembrolizumab) for treating certain lung cancer patients. This designation means that review and approval will be accelerated so that the drug can more quickly reach patients in the U.S., outside of clinical trials. Keytruda is an immunotherapy drug, meaning that it boosts a patient’s own immune system to fight cancer. The breakthrough therapy designation specifically applies to patients with advanced non-small cell lung cancer (NSCLC) whose tumors have tested negative for EGFR mutation and ALK rearrangement, and whose disease worsened despite treatment with platinum-based chemotherapy.

“Merck (NYSE:MRK), known as MSD outside the United States and Canada, announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to KEYTRUDA® (pembrolizumab), the company’s anti-PD-1 therapy, for the treatment of patients with Epidermal Growth Factor Receptor (EGFR) mutation-negative, and Anaplastic Lymphoma Kinase (ALK) rearrangement-negative non-small cell lung cancer (NSCLC) whose disease has progressed on or following platinum-based chemotherapy. This is the second Breakthrough Therapy Designation granted for KEYTRUDA.

“ ‘The FDA’s Breakthrough Therapy Designation of KEYTRUDA underscores that new treatment approaches for advanced non-small cell lung cancer continue to be needed,’ said Dr. Roger Perlmutter, president, Merck Research Laboratories. ‘Our data investigating the use of KEYTRUDA in this difficult-to-treat malignancy are very encouraging, and we look forward to working closely with the FDA to expedite our clinical program.’ ”


The FDA Gives an Ariad Drug Candidate ‘Breakthrough Therapy Designation’

The gist: A new lung cancer treatment called AP26113 has shown promise for certain people with metastatic non-small cell lung cancer (NSCLC). Specifically, it is meant to treat people whose tumors have mutations in the ALK gene and who are resistant to the standard drug crizotinib. It has been tested in volunteer patients in clinical trials. The U.S. Food and Drug Administration (FDA) has now granted breakthrough therapy designation for AP26113, meaning that review and approval will be accelerated so that the drug can more quickly reach patients in the U.S., outside of clinical trials.

“Ariad Pharmaceuticals Inc. of Cambridge said Thursday that federal regulators have granted “breakthrough therapy designation” to a drug candidate for a form of lung cancer.

“According to the Food and Drug Administration’s website, a breakthrough therapy designation means that the FDA will expedite the development and review of such drug. The designation is given to drug candidates designed to treat a serious or life threatening disease that have shown great promise based on preliminary clinical evidence.

“Ariad’s drug candidate, currently dubbed AP26113, is for the treatment of patients with anaplastic lymphoma kinase positive metastatic non-small cell lung cancer who are resistant to crizotinib, an existing treatment on the market.

“Ariad’s press release cited data from the American Cancer Society. According to the society, non-small cell lung cancer is the most common form of lung cancer, accounting for about 85 percent of the estimated 228,190 new cases of lung cancer diagnosed each year in the United States.”