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Regulatory Affairs

PlasmaTech's lead product candidates get FDA orphan drug and rare pediatric disease designations PlasmaTech Biopharmaceuticals (PTBI), a biopharmaceutical company focused on gene therapy and plasma-based products for severe and life- threatening rare diseases announced that the US Food and Drug Administration (FDA) had granted both Orphan Drug Designation and Rare Pediatric Disease Designation for both of PlasmaTech Biopharmaceuticals' lead product candidates for the treatment of Sanfilippo Syndromes A and B.
Regulatory Affairs > News
Bristol-Myers gets amended FDA breakthrough therapy status for daclatasvir-based HCV regimen By PBR Staff Writer
The US Food and Drug Administration (FDA) has amended a previously granted breakthrough therapy designation for Bristol-Myers Squibb's investigational daclatasvir and sofosbuvir combination to treat patients with hepatitis C (HCV).
Regulatory Affairs > News FDA grants orphan drug status for Alnylam's Revusiran to treat TTR-mediated amyloidosis By PBR Staff Writer
The US Food and Drug Administration (FDA) has granted orphan drug designation to Alnylam Pharmaceuticals' investigational RNAi therapeutic, revusiran, to treat transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis).
Regulatory Affairs > News
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Sarepta to submit rolling NDA for eteplirsen to treat DMD
By PBR Staff Writer
US-based biopharmaceutical firm Sarepta Therapeutics has held a pre-new drug application (NDA) meeting with the US Food and Drug Administration (FDA) regarding its lead product candidate, eteplirsen, to treat duchenne muscular dystrophy (DMD).
Regulatory Affairs > News
Boehringer gets acceptance in Scotland for Trajenta-insulin combination to treat T2D
By PBR Staff Writer
The Scottish Medicines Consortium (SMC) has accepted Boehringer Ingelheim's Trajenta (linagliptin) in combination with insulin to treat adults with type 2 diabetes (T2D) to improve glycaemic control.
Regulatory Affairs > News
Therabron's CG100 gets EMA orphan drug status to prevent bronchopulmonary dysplasia
By PBR Staff Writer
US-based biotechnology firm Therabron Therapeutics has received orphan drug designation from the European Medicines Agency (EMA) for its lead product candidate, CG100, for the prevention of bronchopulmonary dysplasia (BPD).
Regulatory Affairs > News
Fibrocell gets FDA rare pediatric disease designation for FCX-007 to treat RDEB
Fibrocell Science (FCSC), an autologous cell and gene therapy company primarily focused on developing first-in-class treatments for rare and serious skin and connective tissue diseases with high unmet medical needs, announced that the US Food and Drug Administration (FDA) has granted rare pediatric disease designation for Fibrocell's lead orphan gene-therapy drug candidate, FCX-007, for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
Regulatory Affairs > News
FDA grants fast track status for Merck's pancreatic cancer drug evofosfamide
By PBR Staff Writer
The US Food and Drug Administration (FDA) has granted fast track designation for Merck's evofosfamide to treat patients living with advanced pancreatic cancer.
Regulatory Affairs > News
Hemispherx Biopharma gets EMA orphan medicine designation for Ampligen to treat Ebola Virus Disease
Hemispherx Biopharma has announced that its European subsidiary, Hemispherx Biopharma Europe, received formal notification from the European Commission [EU/3/15/1480-EMA/OD/310/14] approving its Orphan Medicinal Product Application for Ampligen(R), an experimental therapeutic, to treat Ebola Virus Disease (EVD).
Regulatory Affairs > News
FDA grants QIDP and fast track status for Cidara's antifungal product candidate CD101 IV
Cidara Therapeutics, a biotechnology company developing novel anti-infectives and immunotherapies to treat fungal and other infections, announced that the US Food and Drug Administration (FDA) has designated the company’s lead antifungal product candidate, CD101 IV, as a Qualified Infectious Disease Product (QIDP) with Fast Track status.
Regulatory Affairs > News
Effective Supply Chain Mapping Studies
As a provider of monitoring and validation systems for regulated environments, Vaisala regulatory experts are often asked to help troubleshoot issues in pharmaceutical and medical device supply chains. In this article we answer three questions we often receive on how to perform mapping studies for regulated environments.
Regulatory Affairs > Press Releases
AbbVie gets FDA breakthrough therapy status for venetoclax to treat CLL
By PBR Staff Writer
AbbVie has secured breakthrough therapy designation from the US Food and Drug Administration (FDA) for its investigational medicine venetoclax to treat chronic lymphocytic leukemia (CLL) in previously treated (relapsed/refractory) patients with the 17p deletion genetic mutation.
Regulatory Affairs > News
Retrophin gets FDA orphan drug status for RE-024 to treat PKAN
By PBR Staff Writer
US-based pharmaceutical firm Retrophin has received orphan drug from the US Food and Drug Administration (FDA) for RE-024 to treat pantothenate kinase-associated neurodegeneration (PKAN), a rare and lethal autosomal recessive neurodegenerative disorder.
Regulatory Affairs > News
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