PDUFA date expires with no action on Rinvoq. The FDA will not comply with the Prescription Drug User Fee Act (PDUFA) action dates for the additional New Drug Applications (sNDAs) for Rinvoq (upadacitinib) for the treatment of adults with active psoriatic arthritis and adults with active ankylosing spondylitis. The FDA cited the ongoing review of Pfizer’s post-marketing study, ORAL Surveillance, evaluating tofacitinib in patients with rheumatoid arthritis.
No formal regulatory action has been taken regarding the sNDAs for Rinvoq in psoriatic arthritis and ankylosing spondylitis.
Janssen submits NDA for use of Xarelto in children. The Janssen Pharmaceutical Companies of Johnson & Johnson has filed a New Drug Application (NDA) with the FDA for the use of Xarelto (rivaroxaban) in pediatric patients. The NDA targets two pediatric indications: treatment of venous thromboembolism (VTE) and reduction of the risk of recurrent VTE in patients aged from birth to less than 18 years; and thromboprophylaxis in patients 2 years of age and older with congenital heart disease who have undergone the Fontan procedure.
The application is based on data from studies of Xarelto in adults and on data from two Phase 3 clinical studies in pediatric populations: EINSTEIN-Jr and UNIVERSE. The EINSTEIN-Jr study, published in the January issue of The Lancet Haematology, found that children treated with rivaroxaban had a low risk of recurrence and a reduced thrombotic burden without increased bleeding compared to standard anticoagulants.
The UNIVERSE trial is a two-part phase 3 study examining children ages two to eight who have undergone the Fontan procedure, which is used for children born with congenital heart disease that diverts blood flow from the lower body to the lungs. Thrombosis can be a complication of this procedure.
Bayer is filing for combination cancer. Bayer has submitted an additional new drug application (sNDA) to the FDA and a marketing authorization application to the European Medicines Agency (EMA) for approval of the investigational combination of the cancer treatments Aliqopa (copanlisib) and rituximab.
The US filing is for the treatment of patients with relapsed indolent B-cell non-Hodgkin’s lymphoma (iNHL) and is outside the FDA’s accelerated approved indication for the treatment of adult patients with relapsed follicular lymphoma who have received at least two prior systemic therapies. have got . In the EU, Bayer has applied for the treatment of recurrent marginal zone lymphoma, a subtype of iNHL, and the application has been accepted. The submissions are supported by positive results from the phase 3 study CHRONOS-3, which was published in The Lancet Oncology in April 2021.
Anylam Pharmaceuticals Files NDA for Rare Disease Therapy. The FDA has accepted Anylam Pharmaceuticals’ New Drug Application for vutrisiran, an investigational RNAi drug for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The FDA has set an action date of April 14, 2022, under the Prescription Drug User Fee Act (PDUFA).
The application was based on the results of the Phase 3 HELIOS-A trial, in which vutrisiran achieved primary and all secondary endpoints at nine months, with statistically significant improvements in neuropathy, quality of life (QoL) and walking speed, compared to placebo . The data presented by researchers, presented at the 2021 American Academy of Neurology (AAN) Virtual Annual Meeting, showed that in the majority of patients, vutrisiran reversed the manifestations of polyneuropathy with improvements in neuropathy. Vutrisiran also showed an encouraging safety profile with no drug-related discontinuations or deaths.
Vutrisiran has received the Orphan Drug Designation as well as a Fast-Track designation in the United States for the treatment of the polyneuropathy of hATTR amyloidosis in adults.
Prostate cancer drug achieves breakthrough therapy designation. The FDA has awarded the Breakthrough Therapy Designation (BTD) to Novartis’ investigational targeted radiotherapy, 177Lu-PSMA-617, for the treatment of patients with metastatic castration-resistant prostate cancer (mCRPC).
The designation is based on positive data from the pivotal phase 3 VISION study, which showed that the targeted radioligand therapy plus standard of care (SOC) significantly improved overall survival and radiographic progression-free survival for men with progressive PSMA-positive mCRPC2 compared to the SOC alone .
The therapy is being developed by Advanced Accelerator Applications, a Novartis company.
Another Biogen/Eisai Alzheimer’s Therapy gets the Breakthrough designation. The FDA has awarded the Breakthrough Therapy Designation for lecanemab (BAN2401), an anti-amyloid beta (Aβ) protofibril antibody under investigation for the treatment of Alzheimer’s disease.
The designation is based on the recently published results of a phase 2b clinical trial (study 201) of 856 patients with mild cognitive impairment due to Alzheimer’s disease and mild Alzheimer’s disease. The proof-of-concept study 201 examined the impact of lecanemab treatment on brain amyloid beta (Aβ) reduction and clinical decline.
Lilly’s Alzheimer’s Therapy is labeled Breakthrough Therapy. The FDA has awarded the Breakthrough Therapy designation to donanemab, Lilly’s investigational antibody that targets a modified form of beta-amyloid called N3pG. Lilly plans to submit a biological license application for donanemab later this year under the accelerated approval process based on data from TRAILBLAZER-ALZ. The safety, tolerability, and efficacy of donanemab are also being evaluated in the ongoing, randomized, placebo-controlled, double-blind, multicenter Phase 3 study TRAILBLAZER-ALZ 2.
Data from the TRAILBLAZER-ALZ phase 2 trial was recently published in the New England Journal of Medicine. Researchers in this study found that at 76 weeks, donanemab resulted in a better composite score for cognition and ability to perform activities of daily living than placebo, although results for secondary outcomes were mixed.
Research Therapy for Lung Cancer Gets the Breakthrough Designation. The FDA has assigned the Breakthrough Therapy designation to adagrasib, a therapy for the treatment of patients with non-small cell lung cancer (NSCLC) who carry the KRASG12C mutation after previous systemic therapy. This is supported by results from the pivotal Phase 1/2 KRYSTAL-01 study in patients with advanced NSCLC, whose cancer had progressed after prior treatment with immunotherapy and/or chemotherapy. Adagrasib is being developed by Mirati Therapeutics.
FDA Approves Generic Version of COPD Therapy Brovana. The FDA has approved Cipla’s abbreviated New Drug Application (ANDA) for arformoterol tartrate inhalation solution, a generic equivalent of Brovana from Sunovion Pharmaceuticals for the maintenance treatment of bronchoconstriction in patients with chronic obstructive pulmonary disease (COPD), including chronic bronchitis and emphysema. The therapy is now available.
Teva launches generic version of Performomist. Teva Pharmaceuticals has launched the first generic version of Perforomist twice daily, formoterol fumarate inhalation solution, 20 mcg/2 ML, indicated for the treatment of bronchoconstriction in patients with chronic obstructive pulmonary disease (COPD), including chronic bronchitis and emphysema.
Formoterol fumarate inhalation solution is a long-acting beta2-adrenergic agonist (LABA) used to control the symptoms of COPD in adults with COPD. COPD is a chronic lung disease that includes chronic bronchitis, emphysema, or both. It is for use with a nebulizer only.