Fast track

FDA Grants ARX788 Fast Track Designation for HER2-positive Metastatic Breast Cancer

Retrieved on: 
Monday, January 4, 2021
Clinical medicine, Medicine, Health, Antibody-drug conjugates, Breakthrough therapy, Fast track, Food and Drug Administration, HER2/neu, Breast cancer, Trastuzumab deruxtecan, Margetuximab

SAN DIEGO, Jan. 4, 2021 /PRNewswire/ --Ambrx announced that the U.S. Food and Drug Administration (FDA) granted ARX788 Fast Track Designation as monotherapy for the treatment of advanced or metastatic HER2-positive breast cancer patients who have received one or more prior anti-HER2 based regimens in the metastatic setting.

Key Points: 
  • SAN DIEGO, Jan. 4, 2021 /PRNewswire/ --Ambrx announced that the U.S. Food and Drug Administration (FDA) granted ARX788 Fast Track Designation as monotherapy for the treatment of advanced or metastatic HER2-positive breast cancer patients who have received one or more prior anti-HER2 based regimens in the metastatic setting.
  • This designation was granted based on the phase 1 studies that assessed the safety, tolerability, pharmacokinetic (PK), and preliminary efficacy of ARX788.
  • Fast Track Designation is designed by the FDA to facilitate the development and expedite the review of drugs to treat serious conditions that have the potential to address unmet medical needs.
  • The designation enables early and frequent communication with the FDA and is intended to accelerate drug approval and patient access to novel treatment options.

Orphazyme provides regulatory update on arimoclomol for NPC

Retrieved on: 
Sunday, December 27, 2020
Health sciences, Health, Pharmaceuticals policy, Food and Drug Administration, United States Public Health Service, Orphan drug, Breakthrough therapy, New Drug Application, European Medicines Agency, Fast track, Priority review

Orphazyme is working closely with the FDA to support the final review of the new drug application for arimoclomol, saidMolly Painter, US President, Orphazyme.

Key Points: 
  • Orphazyme is working closely with the FDA to support the final review of the new drug application for arimoclomol, saidMolly Painter, US President, Orphazyme.
  • We have responded to all FDA information requests and submitted all outstanding information regarding the arimoclomol NDA for NPC, said Thomas Blaettler, Chief Medical Officer, Orphazyme.
  • Arimoclomol has received FDA Fast-Track and Breakthrough Therapy Designations for NPC, as well as Orphan Drug and Rare Pediatric Disease Designations.
  • In November 2020, the company also submitted a Marketing Authorisation Application to the European Medicines Agency for arimoclomol in NPC.

COVID-19 Clinical Programme Update

Retrieved on: 
Friday, December 18, 2020
Clinical trials, Medical statistics, National Health Service, National Institute for Health Research, Clinical endpoint, Fast track, Health, Articles, Life sciences

The primary assessment of efficacy will be supported by the key secondary endpoint of progression to severe disease or death and other secondary endpoints.

Key Points: 
  • The primary assessment of efficacy will be supported by the key secondary endpoint of progression to severe disease or death and other secondary endpoints.
  • The SG018 trial was deemed an Urgent Public Health study and recognised as a National Priority by the National Institute for Health Research (NIHR) in October 2020.
  • Furthermore, the FDA has awarded SNG001 Fast Track status, enhancing the ability of Synairgen to interact with the FDA and shortening review timelines.
  • After a slow start in the summer, recruitment and dosing are now progressing well with a noticeable increase in the last two months.

Novaremed Receives Fast Track Designation from the FDA for NRD135S.E1 for the Treatment of Painful Diabetic Peripheral Neuropathy

Retrieved on: 
Friday, December 18, 2020
Health, Diabetes, Clinical trials, Research, Science, Pharmaceutical, Biotechnology, Food and Drug Administration, Health care, Health sciences, Life sciences, Fast track, Breakthrough therapy, Priority review, Fast track, Accelerated approval

Novaremed AG (Novaremed), a clinical-stage Swiss biopharmaceutical company, announced today that it has received Fast Track Designation (FTD) from the U.S. Food and Drug Administration (FDA) for the development of NRD135S.E1 for the treatment of Painful Diabetic Peripheral Neuropathy (PDPN).

Key Points: 
  • Novaremed AG (Novaremed), a clinical-stage Swiss biopharmaceutical company, announced today that it has received Fast Track Designation (FTD) from the U.S. Food and Drug Administration (FDA) for the development of NRD135S.E1 for the treatment of Painful Diabetic Peripheral Neuropathy (PDPN).
  • By awarding Fast Track Designation to NRD135S.E1, an investigational candidate for the treatment of PDPN, the FDA has recognized its potential to meaningfully improve outcomes for patients with this debilitating disease.
  • Fast Track Designation is an FDA process designed to facilitate the development, and expedite the review of, medicines to treat serious conditions and fill unmet medical need.
  • Fast Track Designation can lead to an Accelerated Approval and Priority Review, if certain criteria are met.

COVID-19 Clinical Programme Update

Retrieved on: 
Friday, December 18, 2020
Clinical trials, Medical statistics, National Health Service, National Institute for Health Research, Clinical endpoint, Fast track, Health, Articles, Life sciences

The primary assessment of efficacy will be supported by the key secondary endpoint of progression to severe disease or death and other secondary endpoints.

Key Points: 
  • The primary assessment of efficacy will be supported by the key secondary endpoint of progression to severe disease or death and other secondary endpoints.
  • The SG018 trial was deemed an Urgent Public Health study and recognised as a National Priority by the National Institute for Health Research (NIHR) in October 2020.
  • Furthermore, the FDA has awarded SNG001 Fast Track status, enhancing the ability of Synairgen to interact with the FDA and shortening review timelines.
  • After a slow start in the summer, recruitment and dosing are now progressing well with a noticeable increase in the last two months.

DURECT Corporation Announces DUR-928 Granted FDA Fast Track Designation for Treatment of Alcoholic Hepatitis

Retrieved on: 
Wednesday, December 16, 2020
Fast track, Life sciences, Food and Drug Administration, Investigational New Drug, Fast track, Health

CUPERTINO, Calif., Dec.16, 2020 /PRNewswire/ -- DURECT Corporation (Nasdaq: DRRX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to DUR-928 for the treatment of alcoholic hepatitis (AH, also known as alcohol-associated hepatitis).

Key Points: 
  • CUPERTINO, Calif., Dec.16, 2020 /PRNewswire/ -- DURECT Corporation (Nasdaq: DRRX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to DUR-928 for the treatment of alcoholic hepatitis (AH, also known as alcohol-associated hepatitis).
  • "Fast Track Designation highlights the life-threatening nature of AH and the lack of therapeutic options for this devastating condition," said Norman L. Sussman, M.D., Chief Medical Officer of DURECT Corporation.
  • The FDA grants Fast Track Designation to facilitate development and expedite the review of therapies with the potential to treat a serious condition where there is an unmet medical need.
  • DUR-928 and POSIMIR are investigational drug candidates under development and have not been approved for commercialization by theU.S.

HemoShear Therapeutics' HST5040 Receives U.S. Orphan Drug Designations for Treatment of Methylmalonic Acidemia and Propionic Acidemia

Retrieved on: 
Wednesday, December 16, 2020
Rare diseases, Health, Life sciences, Orphan drug, Propionic acidemia, Methylmalonic acidemia, Fast track

CHARLOTTESVILLE, Va., Dec. 16, 2020 /PRNewswire/ --HemoShear Therapeutics, a clinical stage company developing treatments for rare metabolic disorders, is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designations to HemoShear's HST5040 oral small molecule for treatment of methylmalonic acidemia (MMA) and propionic acidemia (PA).

Key Points: 
  • CHARLOTTESVILLE, Va., Dec. 16, 2020 /PRNewswire/ --HemoShear Therapeutics, a clinical stage company developing treatments for rare metabolic disorders, is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designations to HemoShear's HST5040 oral small molecule for treatment of methylmalonic acidemia (MMA) and propionic acidemia (PA).
  • HST5040 was granted Fast Track and Rare Pediatric Disease designations to treat MMA and PA earlier in 2020.
  • Orphan Drug Designation is awarded by the FDA for therapies to treat diseases that affect fewer than 200,000 people in the U.S.
  • HST5040 is designed for convenient daily administration at home as a liquid formulation taken either orally or through a gastric feeding tube.

Fortress Biotech Announces Breakthrough Therapy Designation for CUTX-101, Copper Histidinate, for the Treatment of Menkes Disease

Retrieved on: 
Tuesday, December 15, 2020
Health, Clinical medicine, Rare diseases, Inborn errors of metal metabolism, Food and Drug Administration, Menkes, Breakthrough therapy, ATP7A, Fast track, Orphan drug, Occipital horn syndrome, Priority review

The FDA previously granted Orphan Drug, Fast Track, and Rare Pediatric Disease Designations to CUTX-101 for the treatment of Menkes disease.

Key Points: 
  • The FDA previously granted Orphan Drug, Fast Track, and Rare Pediatric Disease Designations to CUTX-101 for the treatment of Menkes disease.
  • We are very pleased that the FDA has granted Breakthrough Therapy Designation to CUTX-101, a devastating pediatric disease with no FDA-approved treatment options currently available.
  • Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or life-threatening conditions.
  • Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of copper transporter ATP7A.

Gannex Received U.S. FDA Fast Track Designation for Its NASH Drug Candidate ASC42，an FXR Agonist

Retrieved on: 
Monday, December 14, 2020
Branches of biology, Life sciences, Biology, Receptor agonists, Transcription factors, Pharmacodynamics, Farnesoid X receptor, FXR, Fast track, Agonist, Food and Drug Administration, Tropifexor

This Fast Track designation represents FDA's recognition of ASC42's potential in addressing these unmet medical needs for NASH patients.

Key Points: 
  • This Fast Track designation represents FDA's recognition of ASC42's potential in addressing these unmet medical needs for NASH patients.
  • Gannex received the investigational new drug application (IND) approval for ASC42 from the U.S. FDA in October this year.
  • ASC42is an in-house developednovel non-steroidal, selective, potent Farnesoid X Receptor (FXR) agonist with best-in-class potential.
  • "We are thrilled that the FDA granted Fast Track designation for our FXR agonist ASC42 which is discovered and developed by our in-house talented R&D team," said Dr. Jinzi J. Wu, "This critical recognition by FDA will accelerate global development of ASC42, a potential best-in-class FXR agonist."

Lexicon Pharmaceuticals Receives Fast Track Designation From the FDA for LX9211 for Diabetic Peripheral Neuropathic Pain

Retrieved on: 
Friday, December 11, 2020
Nervous system, Branches of biology, Organ systems, Pain, Analgesics, Neuropathic pain, Calcium channel blockers, Peripheral neuropathy, Fast track, Postherpetic neuralgia, Mirogabalin, Palmitoylethanolamide

THE WOODLANDS, Texas, Dec. 11, 2020 (GLOBE NEWSWIRE) -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), announced today that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the development of LX9211 in diabetic peripheral neuropathic pain.

Key Points: 
  • THE WOODLANDS, Texas, Dec. 11, 2020 (GLOBE NEWSWIRE) -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), announced today that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the development of LX9211 in diabetic peripheral neuropathic pain.
  • The FDAs Fast Track designation of LX9211 reflects the serious unmet medical need of people suffering from diabetic peripheral neuropathic pain, said Praveen Tyle, Ph.D., executive vice president of research and development.
  • Lexicon is currently enrolling patients with diabetic peripheral neuropathic pain in a Phase 2 proof-of-concept study of LX9211 and is preparing to initiate a second Phase 2 clinical trial of LX9211 in post-herpetic neuralgia.
  • Programs receiving Fast Track designation may benefit from early and frequent interactions with the FDA over the course of drug development.