All The Most Important Pharmaceutical News Stories Of The Week – September 2018 – Week # 1
Novartis receives European Commission approval of its CAR-T cell therapy, Kymriah® (tisagenlecleucel)
August 27, 2018
Novartis has announced that the European Commission (EC) has approved Kymriah® (tisagenlecleucel, formerly CTL019). The approved indications are for the treatment of pediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse; and for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. Kymriah developed in collaboration with the University of Pennsylvania (Penn) is a ground-breaking one-time treatment that uses a patient’s own T cells to fight cancer, and the only chimeric antigen receptor T cell (CAR-T) therapy to receive regulatory approval in the EU for these two distinct B-cell malignancies. Kymriah was also the first CAR-T cell therapy ever approved by the US Food and Drug Administration (FDA).
Pfizer Terminates Domagrozumab (Pf-06252616) Clinical Studies for The Treatment of Duchenne Muscular Dystrophy
August 30, 2018
Pfizer Inc. has announced that it is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy (DMD): a Phase 2 safety and efficacy study (B5161002) and an open-label extension study (B5161004). The Phase 2 study (B5161002), did not meet its primary efficacy endpoint, which was to demonstrate a difference in the mean change from baseline in 4 Stair Climb (in seconds) following one year of treatment with domagrozumab as compared to placebo in patients with DMD. Further evaluation of the totality of evidence including secondary endpoints did not support a significant treatment effect. The decision comes after a thorough review of data available at the time of the primary analysis, which evaluated all study participants after one year of treatment, as well as those participants who were in the trial beyond one year. The studies were not terminated for safety reasons. Pfizer will continue to review the data to better understand any insights they may provide, and will share results with the scientific and patient community.
European Commission approves Novartis combination therapy Tafinlar® + Mekinist® for adjuvant treatment of BRAF V600 mutation-positive melanoma
August 29, 2018
Novartis announced today that the European Commission (EC) has approved Tafinlar® (dabrafenib) in combination with Mekinist® (trametinib) for the adjuvant treatment of stage III patients with BRAF V600 mutation-positive melanoma after complete surgical resection. This approval is the third for Tafinlar in combination with Mekinist in Europe across a variety of tumor types identified with a high level of BRAF mutation.
European Commission Approves BLINCYTO® (blinatumomab) For Use In Pediatric Patients With Philadelphia Chromosome-Negative Relapsed Or Refractory B-Cell Precursor Acute Lymphoblastic Leukemia
August 29, 2018
Amgen has announced that the European Commission (EC) has approved an expanded indication for BLINCYTO® (blinatumomab) as monotherapy for the treatment of pediatric patients aged one year or older with Philadelphia chromosome-negative CD19 positive B-cell precursor acute lymphoblastic leukemia (ALL), which is refractory or in relapse after receiving at least two prior therapies or in relapse after receiving prior allogeneic hematopoietic stem cell transplantation. The approval is based on results from the Phase 1/2 ‘205 study, an open-label, multicenter, single-arm trial which evaluated the efficacy and safety of BLINCYTO in pediatric patients with relapsed or refractory B-cell precursor ALL.
FDA Approves Merck’s DELSTRIGO™ (doravirine / lamivudine / tenofovir disoproxil fumarate), a Once-Daily Fixed-Dose Combination Tablet as a Complete Regimen and PIFELTRO™ (doravirine), an NNRTI, Both for the Treatment of HIV-1 in Appropriate Patients
August 30, 2018
Merck, known as MSD outside the United States and Canada, has announced that the U.S. Food and Drug Administration (FDA) has approved two new HIV-1 medicines: DELSTRIGO™, a once-daily fixed-dose combination tablet of doravirine (100 mg), lamivudine (3TC, 300 mg) and tenofovir disoproxil fumarate (TDF, 300 mg); and PIFELTRO™ (doravirine, 100 mg), a new non-nucleoside reverse transcriptase inhibitor (NNRTI) to be administered in combination with other antiretroviral medicines. Both DELSTRIGO and PIFELTRO are indicated for the treatment of HIV-1 infection in adult patients with no prior antiretroviral treatment experience, and are administered orally once daily with or without food. DELSTRIGO contains a boxed warning regarding post-treatment acute exacerbation of hepatitis B (HBV) infection. DELSTRIGO and PIFELTRO do not cure HIV-1 infection or AIDS.
European Commission approves new easy-to-use, once-weekly Bydureon BCise device for patients with type-2 diabetes
August 30, 2018
AstraZeneca has announced that the European Commission (EC) has approved Bydureon BCise (exenatide 2mg prolonged-release suspension for injection in pre-filled pen) as a new formulation within the marketing authorisation for Bydureon (exenatide extended release) for the treatment of patients with type-2 diabetes.
Bayer receives US approval for modern hemophilia A treatment Jivi® with a step-wise prophylaxis dosing regimen
August 30, 2018
Bayer announced that the United States (U.S.) Food and Drug Administration (FDA) has approved Jivi® (BAY94-9027) for the routine prophylactic treatment of hemophilia A in previously treated adults and adolescents 12 years of age or older in the U.S. The recommended initial prophylactic regimen for Jivi is twice weekly, with the ability to dose every five days and further individually adjust to less or more frequent dosing based on bleeding episodes. The FDA also approved Jivi for on-demand treatment and the perioperative management of bleeding in the same population. This approval is based on results from the Phase 2/3 PROTECT VIII trial. Jivi is the third FDA-approved hemophilia A treatment in Bayer’s hemophilia portfolio.
August 31, 2018
AstraZeneca and MedImmune, its global biologics research and development arm, has announced top-line results from the TULIP 1 Phase III trial for anifrolumab in adult patients with moderate-to-severe systemic lupus erythematosus (SLE). The trial did not meet the primary endpoint of a statistically-significant reduction in disease activity in patients with SLE as measured by the SLE Responder Index 4 (SRI4) at 12 months.Top Pharma News of the Week – September 3rd last edit: 2018-09-04T19:42:04+00:00 da