Insights+: The US FDA New Drug Approvals in September 2020

The US FDA has approved 6 NDAs in Sept 2020, leading to treatments for patients and advances in the health care industry. The Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) have approved 85 novel products so far in 2020, including 6 in Sept 2020. Additionally, last year in 2019, the US FDA has approved 48 novel products. We have compiled a list of a total of 6 new drugs approved by the US FDA in Sept 2020.

1. Athena Bioscience’s Qdolo (tramadol hydrochloride) Received the US FDA’s NDA Approval for Oral Solution

Published: Sept 01, 2020 | Tags: Athena Bioscience, QDOLO, tramadol hydrochloride, Receives, U.S. FDA, Approval, NDA, Oral, Solution

  • Athena Biosciences announced that the US FDA has approved Qdolo (tramadol hydrochloride) oral solution 5mg/1mL C-IV
  • The most common AE’s (≥15.0%) observed in patients were dizziness/vertigo, nausea, constipation, headache, somnolence, vomiting, and pruritus
  • Qdolo is indicated in adults for the management of pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate. It will be available at the chain, independent, and specialty pharmacies

2. BMS’ Onureg (azacitidine) Received the US FDA’s Approval as Continued Treatment for Acute Myeloid Leukemia

Published: Sept 01, 2020 | Tags: Azacitidine, Acute Myeloid Leukemia, Approval, BMS, Continued Treatment, FDA, Onureg, US

  • The approval is based on P-III QUAZAR AML-001 study assessing Onureg (300 mg, PO) vs PBO  for 14 days of a 28-day cycle + BSC in a ratio (1:1) in 472 patients aged ≥55yrs. with AML and had achieved CR or Cri following intensive induction CT and cannot proceed with intensive curative therapy including HSCT
  • The P-III QUAZAR AML-001 study results: m-OS (24.7 vs 14.8mos.); m-DOR (12 vs 6 cycles). Additionally, the therapy has received the US FDA’s PR designation while EMA validates its MAA in May’2020
  • Onureg (CC-486) is an oral hypomethylating agent that incorporates into DNA and RNA and carries out hypomethylation of DNA, as well as direct cytotoxicity to abnormal hematopoietic cells in the bone marrow

3. RadioMedix and Curium Detectnet (copper Cu 64 dotatate injection) Received the US FDA’s Approval for Positive Neuroendocrine Tumors (NETs)

Published: Sept 03, 2020 | Tags: RadioMedix, Curium, Detectnet, copper Cu 64 dotatate injection, US FDA Approval, Positive Neuroendocrine Tumors (NETs)

  • The approval is based on P-lll study which involves the assessing of Detectnet (copper Cu 64 dotatate injection) in Neuroendocrine Tumors (NETs) patients. Curium expects to launch detectnet immediately with doses available through various nuclear pharmacies
  • The P-III results demonstrated that the clinical sensitivity and specificity provide a great aid to clinicians in develop accurate treatment approach for their NET patients. Additionally, the 12.7-hrs. half-life allows detectnet to be produced centrally and shipped to sites
  • Detectnet is a positron emission tomography (PET) agent indicated for the localization of somatostatin receptor positive neuroendocrine tumors (NETs) in adult patients

4. Genentech’ Gavreto (pralsetinib) Received the US FDA’s Approval Metastatic RET Fusion-Positive Non-Small Cell Lung Cancer

Published: Sept 04, 2020 | Tags: Pralsetinib, Adults, Approval, FDA, Fusion-Positive, Gavreto, Genentech, Metastatic Non-Small Cell Lung Cancer, Rearranged, Transfection, US

  • The approval is based on P-I/II ARROW study assessing Gavreto (400mg, qd) in RET fusion-positive NSCLC patients with or without prior therapy, and regardless of RET fusion partner or CNS involvement. The study also involves patients with RET-MTC, RET fusion-positive thyroid cancer, and other RET-altered solid tumors
  • Results: ORR (57%) and CR rate (5.7%) in 87 NSCLC patients prior to treated with Pt. based CT, m-DOR was not reached, in 27 treatment-naïve NSCLC patients, ORR (70%); CR rate (11%). Gavreto is now the 6th FDA-approved medicine in Genentech’s portfolio for lung cancer and will co-commercialize by Genentech and Blueprint Medicines in the US
  • Gavreto is an oral precision therapy targeting RET alterations, including fusions and mutations, regardless of the tissue of origin. Additionally, the US FDA has granted BT designation for RET fusion+ NSCLC and RET mutation+ MTC and PR designation for advanced or m-RET-mutant MTC and RET fusion+ thyroid cancer to the therapy with its anticipated PDUFA date as Feb 28, 2021

5. Pfizer’s Xeljanz (tofacitinib) Received the US FDA’s Approval for Active Polyarticular Course Juvenile Idiopathic Arthritis

Published: Sept 28, 2020 | Tags: Active Polyarticular Course Juvenile Idiopathic Arthritis, Approval, FDA, Tofacitinib, US, Xeljanz

  • The approval is based on P-III study including 2 phases assessing tofacitinib (5mg or 1mg/mL oral solution, bid) for 44wks. The 2phases includes an 18 wks. open-label, run-in phase study of 225 patients and a 26wks. PBO controlled withdrawal study of 173 patients
  • Results: the study met its 1EP showing ACR30 response at the end of the run-in phase; the occurrence of disease flare in patients (31% vs 55%) @44wks. The approval makes Xeljanz the first and only JAK inhibitor approved in the US for pcJIA
  • The US FDA has approved the two formulations of Xeljanz (a tablet and an oral solution) and are dosed based on weight. The oral solution is expected to be available by the end of Q1’21 while the tablets (5mg) are available instantly

6. Eton Pharmaceutical’s Alkindi Sprinkle (hydrocortisone) Received the US FDA’s Approval as Replacement Therapy in Pediatric Patients

Published: Sept 29, 2020 | Tags: Eton Pharmaceuticals, Alkindi Sprinkle, hydrocortisone, US FDA Approval, Adrenocortical Insufficiency

  • The approval is based on 6 clinical studies, including the 1st and only interventional P-lll study which involves the assessing of Alkindi Sprinkle (hydrocortisone) for Pediatric AI in neonates to children under 8 yrs. of age
  • Eton expects Alkindi Sprinkle to be commercially available in the Q4’ 2020 and it will be available in four strengths: 0.5mg, 1mg, 2mg, and 5mg, to give greater dosing flexibility to clinicians
  • Alkindi Sprinkle is the 1st and only FDA-approved granular hydrocortisone formulation for the treatment of adrenocortical insufficiency specifically designed for use in children

Related Post: Insights+: The US FDA New Drug Approvals in August 2020

The post Insights+: The US FDA New Drug Approvals in September 2020 first appeared on PharmaShots.