Biologics
FDA has issued a complete response letter for Mylan´s BLA for MYL-1401H, a proposed biosimilar of Amgen’s Neulasta (pegfilgrastim). The letter relates to a “pending update of the BLA with certain [chemistry, manufacturing and controls] data from facility requalification activities post recent plant modifications,” according to the
Biologics
FDA has accepted for priority review an Ultragenyx Pharmaceutical and Kyowa BLA for burosumab to treat pediatric and adult patients with X-Linked Hypophosphatemia (XLH). The agency has set a user fee review action target date of 4/17/2018. FDA previously designated burosumab as a “rare pediatric disease” that
Biologics
There were 669 total CBER recall events by class in FY 2017, up 11% from 575 in FY 2016. There were 427 Class 2 recalls spread over nine categories and 185 Class 3 recalls, as well as 57 mixed class (2 and 3) recalls, according to the FY 2017 report.
Biologics
Harvard Medical School researchers say that noninferiority clinical trial designs are being applied more commonly and broadly and when appropriately designed and executed can identify innovative treatment alternatives with clinical value. Writing in the New England Journal of Medicine, the authors say that noninferiority trials “have become a major tool
Biologics
Recent FDA enforcement actions in two cases in California and Florida relating to use of fat/adipose stem cells are raising the question of whether the agency is changing its approach to the notion of such cells as unapproved biological drugs. That’s the view of Paul Knoepfler in a
Biologics
FDA has accepted for review an Adello Biologics BLA for a biosimilar candidate referencing Amgen’s Neupogen (filgrastim), indicated for treating neutropenia. Filgrastim is a short acting granulocyte-colony stimulating factor that increases the body’s production of white blood cells. The BLA submission is supported by similarity data from
Biologics
FDA has published a guidance, Deviation Reporting for Human Cells, Tissues, and Cellular and Tissue-Based Products Regulated Solely Under Section 361 of the Public Health Service Act and 21 CFR Part 1271, with recommendations and relevant examples for establishments that manufacture certain non-reproductive human cells, tissues, and cellular
Biologics
FDA has extended by three months a Biocon BLA for a proposed trastuzumab biosimilar product (Roche’s Herceptin), indicated for HER2-positive breast cancer and gastric cancer. The company said that the agency “has notified our partner Mylan that they will extend the target action date” to 12/3 in
Biologics
FDA has approved a Boehringer Ingelheim Pharmaceuticals BLA for Cyltezo, a biosimilar version of Abbott’s Humira (adalimumab) for multiple inflammatory diseases. Cyltezo is approved for the following indications in adult patients: * moderately to severely active rheumatoid arthritis; * active psoriatic arthritis; * active ankylosing spondylitis (an arthritis that affects the spine)
Biologics
FDA says that Organogeneris has failed to submit a pediatric assessment for its Gintuit (allogenic cultured keratinocytes and fibroblasts in bovine collagen) allogenic oral soft tissue product as required under the Pediatric Research Equity Act (PREA). The need for the study was included in the agency’s 3/9/12
Biologics
FDA has accepted for priority review a Seattle Genetics supplemental BLA for Adcetris (brentuximab vedotin) for use in patients with cutaneous T-cell lymphoma. The submission is based on data from the Phase 3 ALCANZA trial and two phase 2 investigator-sponsored trials. The agency has set a user fee
Biologics
FDA has cautioned a New York doctor that he must stop marketing in the U.S. a technique called spindle nuclear transfer in which the nucleus of a donor egg is removed and the DNA of another woman’s egg is injected. The doctor, John Zhang, founder of New Hope
Biologics
Public Citizen says the HHS Office for Human Research Protections and the Department of Veterans Affairs should immediately suspend the Myocardia Ischemia and Transfusion (MINT) trial that is expected to involve thousands of heart attack patients in the U.S. and Canada, citing alleged serious ethical violations. In an 8/
Biologics
FDA has accepted an Amgen BLA for Aimovig (erenumab) for preventing migraine in patients experiencing four or more migraine days per month. If approved, Aimovig would be the first monoclonal antibody targeting the calcitonin gene-related peptide (CGRP) receptor, the company says. The submission includes data from pivotal studies involving
Biologics
FDA has accepted a CSL Behring BLA for Hizentra [immune globulin subcutaneous (human) 20% liquid] for treating chronic inflammatory demyelinating polyneuropathy (CIDP) as maintenance therapy to prevent relapse of neuromuscular disability and impairment. CIDP is a rare immune-mediated disorder involving peripheral nerves and the effects can worsen over time.
Biologics
BioMarin Pharmaceutical Inc. has filed a BLA for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, indicated for reducing blood phenylalanine (Phe) levels in adult patients with Phenylketonuria (PKU) who have uncontrolled blood Phe levels on existing therapy. “Pegvaliase has been shown to lower blood Phe levels, which was
Biologics
FDA has accepted for review a Theratechnologies BLA for ibalizumab as a treatment for multidrug resistant human immunodeficiency virus-1. The company says that, if approved, ibalizumab will be the first antiretroviral treatment with a new mechanism of action to be introduced in nearly 10 years and the only treatment
Biologics
CBER’s Office of Compliance and Biologics Quality/Division of Inspections and Surveillance says it entered 51,229 deviation reports into the Biological Product Deviation database in FY 2016, up 10% from the number of reports entered in FY 15. Blood and source plasma establishments submitted 4,546 more reports
Biologics
FDA has approved a CSL Behring BLA for Haegarda, the first C1 esterase inhibitor (human) for subcutaneous administration to prevent hereditary angioedema (HAE) attacks in adolescent and adult patients. “The subcutaneous formulation allows patients to administer the product at home to help prevent attacks,” an FDA release says. Haegarda is
Biologics
Biosimilar products are likely to launch faster under a 6/12 U.S. Supreme Court ruling that they do not have to wait until FDA approval to provide a required six-month marketing advance notice. In Sandoz vs. Amgen, the justices ruled that biosimilar applicants may provide notice either before
Biologics
OncBioMune Pharmaceuticals has submitted to FDA a Phase 2 clinical trial protocol for ProscaVax, a novel therapeutic cancer vaccine for early stage prostate cancer. In the trial, ProscaVax will be evaluated for safety, tolerability and efficacy as a treatment for patients with a new disease diagnosis and who have chosen
Biologics
Amgen has submitted a supplemental BLA for Repatha (evolocumab), a PCSK9 inhibitor, based on a cardiovascular outcomes study (FOURIER) which the company says showed that “maximally reducing low-density lipoprotein cholesterol (LDL-C) levels with Repatha, beyond what is possible with the current best therapy alone, leads to a further