Food and Drug Administration

 FDA Grants Orphan Drug Designation to Neurogene’s Gene Therapy for the Treatment of CLN5 Batten Disease

Tuesday, July 7, 2020 - 2:00pm

CLN5 is a devastating neurodegenerative disease with no FDA approved treatment options, said Rachel McMinn, Ph.D., Neurogenes Founder and Chief Executive Officer.

Key Points: 
  • CLN5 is a devastating neurodegenerative disease with no FDA approved treatment options, said Rachel McMinn, Ph.D., Neurogenes Founder and Chief Executive Officer.
  • Receiving Orphan Drug Designation from the FDA is an important regulatory milestone, and we look forward to advancing our gene therapy program into the clinic.
  • The FDA grants Orphan Drug Designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States.
  • The subtype CLN5 is a rare, pediatric-onset and rapidly progressive disease caused by defects in the CLN5 gene.

Santen Announces U.S. FDA Acceptance of Premarket Approval (PMA) Application for DE-128 (MicroShunt) for Review

Tuesday, July 7, 2020 - 12:40am

OSAKA, Japan, July 6, 2020 /PRNewswire/ -- Santen Pharmaceutical Co., Ltd. (hereinafter, Santen) today announced that the United States Food and Drug Administration (FDA) has accepted the Premarket Approval (PMA) application for DE-128 (MicroShunt) for review.

Key Points: 
  • OSAKA, Japan, July 6, 2020 /PRNewswire/ -- Santen Pharmaceutical Co., Ltd. (hereinafter, Santen) today announced that the United States Food and Drug Administration (FDA) has accepted the Premarket Approval (PMA) application for DE-128 (MicroShunt) for review.
  • The substantive review, which traditionally spans 180 days from the FDA's receipt of the PMA final module, is an in-depth review following which the FDA will make a decision as to whether the PMA is approved.
  • As a specialized company dedicated to ophthalmology, Santen carries out research, development, marketing, and sales of pharmaceuticals, over-the-counter products, and medical devices.
  • Santen is the market leader for prescription ophthalmic pharmaceuticals in Japan and its products now reach patients in over 60 countries.

Chembio Diagnostics Awarded BARDA Contract for Development of DPP COVID-19 Point-of-Care Antigen System

Monday, July 6, 2020 - 9:10pm

The contract is intended to assist Chembio in developing a COVID-19 point-of-care antigen system using Chembios proprietary DPP technology and requesting a U.S. Food and Drug Administration Emergency Use Authorization (EUA) for the system.

Key Points: 
  • The contract is intended to assist Chembio in developing a COVID-19 point-of-care antigen system using Chembios proprietary DPP technology and requesting a U.S. Food and Drug Administration Emergency Use Authorization (EUA) for the system.
  • Chembio will use the funds under contract number 75A50120C00138 to accelerate development of its DPP COVID-19 Antigen System, which is expected to consist of a DPP COVID-19 Antigen Assay and DPP Micro Reader and to use a respiratory specimen, such as a nasal or nasopharyngeal swab, to detect SARS-CoV-2 antigens.
  • Chembio is a leading point-of-care diagnostics company focused on detecting and diagnosing infectious diseases, including COVID-19, sexually transmitted disease, and fever and tropical disease.
  • Forward-looking statements include statements regarding the intent, belief or current expectations of Chembio and its management with respect to the development of, and obtaining an EUA for, a COVID-19 point-of-care antigen system.

Coronavirus (COVID-19) Update: FDA Issued Emergency Use Authorization for Point of Care Antigen Test

Monday, July 6, 2020 - 7:03pm

SILVER SPRING, Md., July 6, 2020 /PRNewswire/ -- The U.S. Food and Drug Administration issued an Emergency Use Authorization (EUA) for a COVID-19 antigen diagnostic test, the BD (Becton Dickinson) Veritor System for Rapid Detection of SARS-CoV-2.

Key Points: 
  • SILVER SPRING, Md., July 6, 2020 /PRNewswire/ -- The U.S. Food and Drug Administration issued an Emergency Use Authorization (EUA) for a COVID-19 antigen diagnostic test, the BD (Becton Dickinson) Veritor System for Rapid Detection of SARS-CoV-2.
  • This is the second antigen test the FDA has authorized for the detection of SARS-CoV-2 antigens.
  • Emergency use of this test is limited to authorized laboratories using the BD Veritor Plus Analyzer Instrument.
  • Antigen tests play an important role in the overall response against COVID-19, including as a point-of-care test that can potentially scale up to test millions of Americans quickly," said FDA Commissioner Stephen M. Hahn, M.D.

AVROBIO Announces New Patients Dosed in Gaucher Disease and Cystinosis Clinical Trials

Monday, July 6, 2020 - 12:00pm

The company also announced that the second patient has been dosed in the ongoing investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 for cystinosis.

Key Points: 
  • The company also announced that the second patient has been dosed in the ongoing investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 for cystinosis.
  • The first patient dosed is an important milestone for the Gaucher disease community and our AVR-RD-02 program.
  • AVROBIO is conducting two clinical trials for its AVR-RD-01 investigational gene therapy for Fabry disease.
  • Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a preclinical program in Pompe disease.

EspeRare Receives FDA Breakthrough Therapy Designation for Investigational Prenatal Treatment ER-004 in X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)

Monday, July 6, 2020 - 12:00pm

These results, demonstrating that this prenatal treatment has a profound and life-changing effect on these infants,werepublished in the New England Journal of Medicine 1 .

Key Points: 
  • These results, demonstrating that this prenatal treatment has a profound and life-changing effect on these infants,werepublished in the New England Journal of Medicine 1 .
  • The FDA Breakthrough Therapy Designation intends to expedite the development and review of drugs for serious or life-threatening conditions.
  • Having the FDA Breakthrough Therapy Designation means scientists can aspire starting a clinical trial to confirm the findings for ER-004 and ultimately, if successful, make this treatment an option for families.
  • In Europe, the program receives support from the EMA's PRIME (Priority Medicines) and also the Orphan Drug Designation.

EspeRare Receives FDA Breakthrough Therapy Designation for Investigational Prenatal Treatment ER-004 in X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)

Monday, July 6, 2020 - 12:00pm

These results, demonstrating that this prenatal treatment has a profound and life-changing effect on these infants,werepublished in the New England Journal of Medicine 1 .

Key Points: 
  • These results, demonstrating that this prenatal treatment has a profound and life-changing effect on these infants,werepublished in the New England Journal of Medicine 1 .
  • The FDA Breakthrough Therapy Designation intends to expedite the development and review of drugs for serious or life-threatening conditions.
  • Having the FDA Breakthrough Therapy Designation means scientists can aspire starting a clinical trial to confirm the findings for ER-004 and ultimately, if successful, make this treatment an option for families.
  • In Europe, the program receives support from the EMA's PRIME (Priority Medicines) and also the Orphan Drug Designation.

ADMA Biologics Opens Its Newest ADMA BioCenters Plasma Collection Facility

Monday, July 6, 2020 - 12:00pm

RAMSEY, N.J. and BOCA RATON, Fla. and KNOXVILLE, Tenn., July 06, 2020 (GLOBE NEWSWIRE) -- ADMA Biologics, Inc. (Nasdaq: ADMA) (ADMA), an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing and developing specialty plasma-derived biologics, today announced the commencement of operations and initiation of collections at its newest ADMA BioCenters plasma collection facility located in Knoxville, TN.

Key Points: 
  • RAMSEY, N.J. and BOCA RATON, Fla. and KNOXVILLE, Tenn., July 06, 2020 (GLOBE NEWSWIRE) -- ADMA Biologics, Inc. (Nasdaq: ADMA) (ADMA), an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing and developing specialty plasma-derived biologics, today announced the commencement of operations and initiation of collections at its newest ADMA BioCenters plasma collection facility located in Knoxville, TN.
  • ADMA also announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval for this second plasma collection center.
  • We are extremely pleased to announce the opening and commencement of operations at our second and newest ADMA BioCenters plasma collection facility, said Adam Grossman, President and Chief Executive Officer of ADMA.
  • ADMA BioCenters is an FDA licensed facility specializing in the collection of human plasma used to make special medications for the treatment and prevention of diseases.

Mylan Secures Regulatory Approval for Remdesivir Lyophilized Powder for Injection 100 mg/vial in India for Restricted Emergency Use in COVID-19 Patients

Monday, July 6, 2020 - 11:00am

Malik continued: "Our approval is a significant milestone for Mylan, for the global public health community and, most importantly, for patients who are battling this pandemic.

Key Points: 
  • Malik continued: "Our approval is a significant milestone for Mylan, for the global public health community and, most importantly, for patients who are battling this pandemic.
  • Mylan previously announced a global collaboration agreement with Gilead Sciencesfor the commercialization of remdesivir in 127 low- and middle-income countries, including India.
  • As a leading global pharmaceutical company, Mylan is committed to continue doing its part in support of public health needs.
  • Based on the results, USFDA granted emergency use authorization (EUA) of Remdesivir to treat hospitalized patients with severe COVID-19 in the U.S.

Medical Devices and COVID-19 - Changes to the Approval Process - ResearchAndMarkets.com

Friday, July 3, 2020 - 3:28pm

There has also been less demand for other medical devices due to a lower number of non COVID-19 related procedures being carried out.

Key Points: 
  • There has also been less demand for other medical devices due to a lower number of non COVID-19 related procedures being carried out.
  • As a result some manufacturers have seen lower demand for their other medical devices and have temporarily closed production facilities and furloughed staff.
  • Analysts expect that the impact of COVID-19 on the medical device industry could lead to greater demand for reshoring of device production and an increased focus on diagnostic devices.
  • In order to avoid shortages of vital medical equipment, approval bodies in the US and European Union have granted priority review and delayed reporting requirements.