Priority review

BeyondSpring Announces Late-Breaking Oral Presentation of Final Phase 3 DUBLIN-3 Data with the Plinabulin/Docetaxel Combination Versus Docetaxel Alone in 2nd/3rd Line Non-Small Cell Lung Cancer Patients with EGFR Wild Type at the European Society for Medi

Retrieved on: 
Tuesday, August 31, 2021
Company, European Society for Medical Oncology, Â, PFS, National Medical Products Administration, ITT, Priority review, Congress, Shanghai Chest Hospital, PD-1, Therapy, Cancer, U.S. Securities and Exchange Commission, EGFR, Large-cell lung carcinoma, Fourth grade, Incidence, GLOBE, Medicine, Respiratory Medicine, Knowledge, CEST, Data, PDUFA, CIN, Form 20-F, Docetaxel, G-CSF, Breakthrough therapy, NDA, NEW, Forward-looking statement, NASDAQ, Safety, APC, B cell, HSPC, Oncology, Patient, NSCLC, Neutropenia, Department, Pharmaceutical industry

This will include the final intention-to-treat (ITT) dataset from the Companys DUBLIN-3 Phase 3 registrational trial of their first-in-class lead asset, plinabulin, in combination with docetaxel vs. docetaxel alone for the treatment of 2nd/3rd line non-small cell lung cancer (NSCLC) patients with EGFR wild type.

Key Points: 
  • This will include the final intention-to-treat (ITT) dataset from the Companys DUBLIN-3 Phase 3 registrational trial of their first-in-class lead asset, plinabulin, in combination with docetaxel vs. docetaxel alone for the treatment of 2nd/3rd line non-small cell lung cancer (NSCLC) patients with EGFR wild type.
  • In the coming months, BeyondSpring plans to apply for an NDA in the U.S. and China for the use of plinabulin in these NSCLC patients.
  • Plinabulin, BeyondSprings lead asset, is a selective immunomodulating microtubule-binding agent (SIMBA), which is a potent antigen presenting cell (APC) inducer.
  • It is a novel, intravenous infused, patent-protected, NDA stage asset for CIN prevention and a Phase 3 anti-cancer candidate for non-small cell lung cancer (NSCLC).

EQS-News: Relief Reports that U.S. Collaboration Partner has Announced a New Finding from Aviptadil Phase 2b/3 Trial Demonstrating Clinically Significant Relief from Respiratory Distress in Critical COVID-19

Retrieved on: 
Tuesday, August 31, 2021
VIP, NAPB, OTCQB, Dark Enlightenment, Priority review, Urea, Failure rate, Acer, Nasdaq, Food, Shortness of breath, Therapy, RLF, SIX Swiss Exchange, Patient, LinkedIn, History, New Drug Application, Maple syrup urine disease, COVID-19, Vasoactive intestinal peptide, Breakthrough therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmaceutical industry

According to the press release, the new analysis shows that patients treated with ZYESAMI demonstrated improvement in blood oxygen, indicative of improved lung function, within a day of starting treatment.

Key Points: 
  • According to the press release, the new analysis shows that patients treated with ZYESAMI demonstrated improvement in blood oxygen, indicative of improved lung function, within a day of starting treatment.
  • This latest analysis also appears to support NRx's plan to submit an application for Breakthrough Therapy Designation to the U.S. Food and Drug Administration for this indication.
  • Relief believes that, if granted, this could confer Priority Review to the aviptadil New Drug Application, when submitted.
  • Relief focuses primarily on clinical-stage programs based on molecules with a history of clinical testing and use in human patients or a strong scientific rationale.

European Commission Approves BioMarin’s VOXZOGO® (vosoritide) for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close

Retrieved on: 
Friday, August 27, 2021
Professional Services, Health, Finance, Banking, Pharmaceutical, Biotechnology, Royal commission, EMA, Priority review, Endochondral ossification, SEC, Research, Food, Ageing, Review, Safety, Radiology, Medicine, ANSM, Otitis, AES, Human, Orphan drug, Sleep apnea, Policy, Use, Adult, Acceleration, European Commission, Puberty, Injection, Degenerative disease, Achondroplasia, Quality of life, FGFR3, Incidence, Skull, Diagnosis, Natural history, ATU, Osteochondrodysplasia, Șaeș, BioMarin Pharmaceutical, CNP, Spine, Asia, BMRN, NASDAQ, Mortality, EC, Hypotension, FDA, Fundacion NYC, View, Spinal stenosis, Company, Face, U.S. Securities and Exchange Commission, Population, Growth, PDUFA, Risk, NDA, Patient, PRV, Classification, Medication, Parent, Pharmaceutical industry, Achondroplasia, BioMarin, ACHONDROPLASIA, BIOMARIN

Achondroplasia is a serious, progressive, and lifelong condition, which can cause multi-system complications that in some cases require surgical intervention.

Key Points: 
  • Achondroplasia is a serious, progressive, and lifelong condition, which can cause multi-system complications that in some cases require surgical intervention.
  • It is estimated that over 11,000 children across Europe, Middle East, and Africa are affected by achondroplasia and could be eligible for treatment with vosoritide.
  • Today, we applaud the European Commission for recognizing the urgent need to treat children with a progressive condition by providing parents of children with achondroplasia with a therapeutic treatment option.
  • Vosoritide is being tested in children whose growth plates are still "open", typically those under 18 years of age.

Avalo Therapeutics, Inc. Announces Corporate Name Change from Cerecor Inc.

Retrieved on: 
Thursday, August 26, 2021
SRD5A3-CDG, Company, Hector Avalos, Private Securities Litigation Reform Act, Nasdaq, Birth, Therapy, U.S. Securities and Exchange Commission, Priority review, Orphan drug, GLOBE, Glycosylation, Microcystic lymphatic malformation, Security (finance), Biomarker, Ulcerative colitis, Food, Forward-looking statement, Probability, Patient, FDA, COVID-19, Lists of diseases, Drug development, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Vaccine, Pharmaceutical industry

In conjunction with the corporate name change, the Company will trade on the Nasdaq Capital Market under the new ticker symbol "AVTX".

Key Points: 
  • In conjunction with the corporate name change, the Company will trade on the Nasdaq Capital Market under the new ticker symbol "AVTX".
  • The new ticker will become effective at the open of the market on August 26, 2021.
  • As we enter this exciting period of our corporate journey, we look forward to the numerous data readouts in the second half the 2021.
  • For more information about Avalo Therapeutics, please visit www.avalotherapeutics.com
    This press release may include forward-looking statements made pursuant to the Private Securities Litigation Reform Act of 1995.

BeyondSpring Announces Exclusive Commercialization and Co-development Agreement with Jiangsu Hengrui Pharmaceuticals for Plinabulin in Greater China

Retrieved on: 
Thursday, August 26, 2021
Company, National Medical Products Administration, Priority review, PD-1, Therapy, Cancer, Global R&D management, U.S. Securities and Exchange Commission, EGFR, Large-cell lung carcinoma, Shanghai Stock Exchange, Medication, GLOBE, Knowledge, COGS, Research, PDUFA, CIN, Director, Food, Form 20-F, Docetaxel, G-CSF, Breakthrough therapy, NDA, Marketing, NEW, Forward-looking statement, NMPA, Board, NASDAQ, Safety, APC, B cell, HSPC, Patient, Partnership, Neutropenia, Neoplasm, FDA, Regulation of tobacco by the U.S. Food and Drug Administration, Fine chemical, Telecommunication, Pharmaceutical industry

Wanchunbulin and Hengrui will co-develop additional indications for plinabulin in the Territory; Hengrui funds 50% of future clinical development costs for new cancer indications in the Territory.

Key Points: 
  • Wanchunbulin and Hengrui will co-develop additional indications for plinabulin in the Territory; Hengrui funds 50% of future clinical development costs for new cancer indications in the Territory.
  • NEW YORK and SHANGHAI, China, Aug. 26, 2021 (GLOBE NEWSWIRE) -- BeyondSpring (the Company or BeyondSpring) (NASDAQ: BYSI), a global pharmaceutical company focused on the development of cancer therapeutics, and Jiangsu Hengrui Pharmaceuticals Co., Ltd. (or Hengrui) today announced an exclusive commercialization and co-development agreement in Greater China (the Territory) for BeyondSpring's investigational drug candidate plinabulin, a first-in-class, selective immunomodulating microtubule-binding agent (SIMBA).
  • BeyondSpring will still retain 100% of the global plinabulin rights outside of Greater China.
  • Under the terms of the agreement, Wanchunbulin will grant Hengrui exclusive rights to commercialize and co-develop plinabulin in the Greater China markets, including mainland China, Hong Kong, Macau and Taiwan.

Servier Announces FDA Approval of TIBSOVO® (ivosidenib tablets) in IDH1-Mutated Cholangiocarcinoma

Retrieved on: 
Wednesday, August 25, 2021
Cancer, PFS, Priority review, IDH1, Progression-free survival, Food, Cholangiocarcinoma, IDH, Shanda, Tablet, Mutation, Review, Achievement, Adult, Enzyme, Patient, Caregiver, CEO, FDA, New Drug Application, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmaceutical industry

The FDA approval of this indication is supported by data from the ClarIDHy study, the first and only randomized Phase 3 trial for previously treatedIDH1-mutated cholangiocarcinoma.

Key Points: 
  • The FDA approval of this indication is supported by data from the ClarIDHy study, the first and only randomized Phase 3 trial for previously treatedIDH1-mutated cholangiocarcinoma.
  • The recommended TIBSOVO dosage for previously treated IDH1-mutated cholangiocarcinoma is 500 mg orally once daily with or without food until disease progression or unacceptable toxicity.
  • "This approval brings new hope to the cholangiocarcinoma community and we are excited that this much-needed new therapeutic option is being made available to patients."
  • Servier Pharmaceuticals is introducing ServierONE Patient Support Services, a program that offers one-on-one support to help patients who are prescribed TIBSOVO or other Servier products navigate their cancer journey.

Enterin Announces Appointment of Hilde Williams as Senior Regulatory Advisor

Retrieved on: 
Wednesday, August 25, 2021
Neurodegeneration, Human, Obesity, Regulatory affairs, Priority review, CNS, Lists of diseases, Parkinson's disease, Infection, ENS, Diabetes, SR, Acadia Pharmaceuticals, CMO, Psychiatry, Enteric nervous system, Neurology, NDA, Alzheimer's disease, Development, Patient, Priority, FDA, Brain, Pharmaceutical industry

PHILADELPHIA, Aug. 25, 2021 /PRNewswire/ -- Enterin, Inc. ,a privately held, Philadelphia-based, clinical-stage biopharmaceutical company pioneering novel treatments for neurodegenerative and metabolic diseases, announcesthat Hilde Williams has joined the organization as a Senior Regulatory Advisor.

Key Points: 
  • PHILADELPHIA, Aug. 25, 2021 /PRNewswire/ -- Enterin, Inc. ,a privately held, Philadelphia-based, clinical-stage biopharmaceutical company pioneering novel treatments for neurodegenerative and metabolic diseases, announcesthat Hilde Williams has joined the organization as a Senior Regulatory Advisor.
  • Williams has successfully led regulatory strategy from Pre-IND through NDA for neurologic, psychiatric, and other development programs and has served in executive leadership team positions.
  • "We are extremely excited to have Hilde lead Regulatory Affairs at Enterin.
  • "I am excited to serve as Senior Regulatory Advisor for Enterin and oversee regulatory strategy and operations for the company's development programs," said Williams.

FDA Approves First COVID-19 Vaccine

Retrieved on: 
Monday, August 23, 2021
Center, Centers for Disease Control and Prevention, Red, Pfizer–BioNTech COVID-19 vaccine, Priority review, Center for Biologics Evaluation and Research, Pain, Infant, Myocarditis, Protein, Immune system, Hospital, RNA, Disease, Food, Research, Death, Pregnancy, Fatigue, Immunodeficiency, EUA, Pfizer, Clinical trial, RNA transfection, Safety, Risk, Pericarditis, Fever, Vaccine, BioNTech, Commissioner of Food and Drugs, Vaccination, FDA, BLA, COVID-19, Headache, Swelling, Chills, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmaceutical industry

"The FDA's approval of this vaccine is a milestone as we continue to battle the COVID-19 pandemic.

Key Points: 
  • "The FDA's approval of this vaccine is a milestone as we continue to battle the COVID-19 pandemic.
  • While this and other vaccines have met the FDA's rigorous, scientific standards for emergency use authorization, as the first FDA-approved COVID-19 vaccine, the public can be very confident that this vaccine meets the high standards for safety, effectiveness, and manufacturing quality the FDA requires of an approved product," said Acting FDA Commissioner Janet Woodcock, M.D.
  • "While millions of people have already safely received COVID-19 vaccines, we recognize that for some, the FDA approval of a vaccine may now instill additional confidence to get vaccinated.
  • For all vaccines, the FDA evaluates data and information included in the manufacturer's submission of a biologics license application (BLA).

U.S. Food and Drug Administration Accepts for Priority Review Bristol Myers Squibb’s Application for Orencia (abatacept) for the Prevention of Acute Graft Versus Host Disease (aGvHD)

Retrieved on: 
Monday, August 23, 2021
Research, Surgery, FDA, Clinical Trials, Cardiology, Stem Cells, Biotechnology, Pharmaceutical, Health, Science, Janus, SOC, Liver, Population, Lymphatic system, Boston Children's Hospital, Priority review, Transplant, TRM, Fibrosis, Bristol Myers Squibb, TNF, Skin, BMY, Infection, CNI, MMUD, Review, PDUFA, Prescription Drug User Fee Act, Food, Doctor of Philosophy, Death, Degenerative disease, RA, GFS, NYSE, Clinical trial, Methotrexate, Gastrointestinal tract, Immunology, Risk, Adult, Disease, HSCT, Patient, Graft-versus-host disease, HLA, MUD, MTX, FDA, Hematopoietic stem cell transplantation, JAK, Dana–Farber Cancer Institute, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmaceutical industry, Dentistry, Vaccine

The FDA granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of December 23, 2021.

Key Points: 
  • The FDA granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of December 23, 2021.
  • Results from ABA2 showed that treatment with Orencia resulted in a significant reduction in severe aGvHD and associated morbidity without an increase in disease relapse.
  • Bristol Myers Squibb thanks the patients and investigators who participated in this clinical trial.
  • It should also be noted that a Priority Review designation does not change the standards for FDA approval.

Senhwa's Silmitasertib Receives US FDA Fast Track Designation for the Treatment of Recurrent Sonic Hedgehog Driven Medulloblastoma

Retrieved on: 
Thursday, August 19, 2021
Human, Priority review, Fast track (FDA), Therapy, Fast Track, Cancer, Consortium, DNA, CK2, Food, Accelerated approval (FDA), RPD, Hospital, Review, Clinical trial, CTEP, US FDA, Patient, SAN, US Food Sovereignty Alliance, SHH, IIT, US, PRV, FDA, Academic health science centre, COVID-19, Drug development, Degenerative disease, Pharmaceutical industry

TAIPEI and SAN DIEGO, Aug. 18, 2021 /PRNewswire/ -- Senhwa Biosciences, Inc. (TPEx: 6492), a drug development company focused on first-in-class therapeutics for oncology, rare diseases, and novel coronaviruses, today announced that the US Food and Drug Administration (FDA) has granted Fast Track Designation for Silmitasertib, a highly selective inhibitor of casein kinase 2 (CK2) to treat patients with recurrent sonic hedgehog (SHH) driven Medulloblastoma.

Key Points: 
  • Fast Track Designation expedites the review of new drugs for serious conditions currently without effective treatments.
  • Through Fast Track, Senhwa is eligible to apply for Accelerated Approval and Priority Review upon reaching relevant criteria with the US FDA.
  • "We are delighted to receive Fast Track Designation and look forward to working closely with the FDA to accelerate the development of Silmitasertib, aiming to promptly bring a meaningful treatment to patients with recurrent SHH driven Medulloblastoma," said Tai-Sen Soong, Chief Executive Officer of Senhwa Biosciences.
  • US FDA granted Silmitasertib an Orphan Drug Designation for the treatment of Cholangiocarcinoma in December 2016 and a Rare Pediatric Disease Drug Designation for the treatment of Medulloblastoma in July 2020.