A Priority Review designation does not alter the scientific/medical standard for approval or the quality of evidence necessary and it does not affect the length of the clinical trial period. Prior to approval, each drug marketed in the United States must go through a detailed FDA review process. FDA drug review time includes two-tiered system […]
Pediatric Rare diseases and Rare Pediatric Disease Designation FDA will award priority review vouchers to sponsors of certain rare pediatric disease product applications that meet the criteria specified. Section 529 of the FD&C Act is intended to encourage development of new drug and biological products for the prevention and treatment of rare pediatric diseases. Section […]
Lesions in the lungs of patients with pneumonia caused by a SARS-CoV-2 infection are distinct from those caused by bacteria. SARS-CoV-2 is known to damage lung tissue as many COVID-19 patients develop pneumonia, which can progress to respiratory failure and sometimes death. In chest CT scans lungs of COVID-19 patients show cloudy lesion patterns that […]
Biotech companies’ continued participation in the Global Orphan Drug Market Opportunity requires the development of new strategies rooted in the established knowledge of orphan developer experts. Participation of big pharmaceutical companies in the ongoing clinical research of orphan drug are about to introduce a drastic change in overall scenario of the approach. Global Orphan Drug […]
The COVID19 has taken the world by a stranglehold and the outbreak is causing significant disruptions in clinical research which is facing significant challenges. The process for conducting conventional clinical trials become difficult and risky at a time when the public is advised to shelter at home and healthcare facilities become overwhelmed with caring for […]
Retrophin’s experimental PKAN drug fosmetpantotenate fails phase III trial Pantothenate kinase-associated neurodegeneration (PKAN) is a rare disease characterized by a progressive neurodegenerative disorder and buildup of iron in the brain which is estimated to affect up to 5000 patients worldwide. PKAN is inherited in an autosomal recessive manner and is caused by mutations in the […]
Biologic treatments show promise in providing clinical solutions to a variety of diseases Due to biological’s unique characteristics, study designers may find that results in pre-clinical trials do not predict acute, chronic and late onset immunogenicity and safety. In Addition, the biological orphan has a long PK half-life of months such as with any mAb, […]
