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Companion Diagnostic Approved for Alpelisib in Metastatic Breast Cancer

The FDA approved FoundationOne CDx to be used as a companion diagnostic for alpelisib in combination with fulvestrant for the treatment of postmenopausal women, and men, with HR+/HER2-, PIK3CA-mutated, advanced or metastatic breast cancer following progression on or after an endocrine-based regimen. It is the first and only FDA-approved broad comprehensive genomic profiling test for all solid tumors, including breast cancer, that incorporates multiple companion diagnostics.

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In May 2019, the FDA approved alpelisib in combination with fulvestrant for the treatment of postmenopausal women, and men, with HR+/HER2-, PIK3CA-mutated, advanced or metastatic breast cancer following progression on or after an endocrine-based regimen metastatic breast cancer with a PIK3CA mutation following progression on or after an endocrine-based regimen. PIK3CA is the most commonly mutated gene in HR+/HER2- breast cancer; approximately 40 percent of patients living with HR+/HER2- breast cancer have this mutation. Professional guidelines were updated in September 2019 to recommend assessment for PIK3CA mutations as part of the workup of HR+/HER2- advanced or metastatic breast cancer.


Atezolizumab With Nab-Paclitaxel & Carboplatin for Metastatic NSCLC Without EGFR/ALK Aberrations

Atezolizumab in combination with paclitaxel protein-bound and carboplatin has been approved for the first-line treatment of adult patients with metastatic non-squamous non-small cell lung cancer (NSCLC) with no EGFR or ALK genomic tumor aberrations.


Efficacy was evaluated in IMpower130 (NCT02367781), a multicenter, randomized (2:1), open-label trial in patients with stage IV non-squamous NSCLC who had received no prior chemotherapy for metastatic disease, but could have received prior EGFR or ALK kinase inhibitor, if appropriate. The trial randomized 724 patients (ITT) to receive atezolizumab, paclitaxel protein-bound, and carboplatin, followed by single-agent atezolizumab or to receive paclitaxel protein-bound and carboplatin, followed by maintenance pemetrexed at the investigator's discretion (control).


The primary efficacy outcome measures were progression-free survival (PFS) by RECIST v1.1 and overall survival (OS) in the subpopulation of patients documented to have no EGFR or ALK genomic tumor aberrations (ITT-WT). In the primary analysis population (ITT-WT, N=681), the estimated median PFS was 7.2 months (95% CI: 6.7, 8.3) for the atezolizumab arm compared to 6.5 months (95% CI: 5.6, 7.4) for the control arm (HR 0.75; 95% CI: 0.63, 0.91; p=0.0024). Median OS was 18.6 months (95% CI: 15.7, 21.1) and 13.9 months (95% CI: 12.0, 18.7), respectively (HR 0.80; 95% CI: 0.64, 0.99; p=0.0384).


The most common adverse reactions (reported in >= 20% of patients) of atezolizumab when administered in combination with other antineoplastic drugs in patients with NSCLC and SCLC were fatigue/asthenia, nausea, alopecia, constipation, diarrhea, and decreased appetite.


The recommended atezolizumab dose for this use is 1,200 mg as an IV infusion every 3 weeks. When atezolizumab is administered on the same day as chemotherapy, atezolizumab should be given first.


Surufatinib Granted FDA Orphan Drug Designation for Pancreatic Neuroendocrine Tumors

The FDA granted Orphan Drug Designation to surufatinib for the treatment of pancreatic neuroendocrine tumors (NET). If approved by the FDA as an orphan treatment, surufatinib will be entitled to 7 years of market exclusivity for the approved indication. Surufatinib is under investigation in multiple solid tumors in China and the U.S., both as a monotherapy and in combination with immunotherapies.


Surufatinib is the second novel oncology drug discovered to successfully complete a phase III trial in China. A New Drug Application for surufatinib for the treatment of patients with advanced non-pancreatic NET was accepted for review by the China National Medical Products Administration.


Regenerative Medicine Advanced Therapy Designation Granted to ADP-A2M4 for Synovial Sarcoma

The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for ADP-A2M4 for the treatment of synovial sarcoma. Earlier this year, FDA granted Orphan Drug Designation to ADP-A2M4 for the treatment of soft tissue sarcomas.


Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products. A product is eligible for RMAT designation if it is a regenerative medicine therapy, such as a T-cell therapy, and is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.


RMAT designation includes the incentives of Breakthrough Therapy Designation, including additional FDA interaction and guidance, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application, and other opportunities to expedite development and review.


Data from patients with synovial sarcoma treated in the expansion phase of a phase I trial with ADP-A2M4 were recently presented. There was an overall response rate of 50 percent, and a disease control rate of 93 percent with 13 out of 14 patients showing clinical benefit with best overall responses of partial responses (confirmed or unconfirmed; n=7) or stable disease (n=6).