Breakthrough therapy

A new FDA Fast Track designation for Zambon

Retrieved on: 
Tuesday, April 28, 2020
Biotechnology, FDA, Pharmaceutical, Health, Health, Articles, Pharmaceutical industry, Fast track, Orphan drugs, Priority review, Food and Drug Administration, New Drug Application, Accelerated approval, Breakthrough therapy, Olaratumab, Zambon S.p.A., Breath Therapeutics, a Zambon company

LCsAi, developed by Breath Therapeutics which was acquired by Zambon in 2019, previously received orphan drug designation from the FDA and European Medicines Agency for the treatment of BOS.

Key Points: 
  • LCsAi, developed by Breath Therapeutics which was acquired by Zambon in 2019, previously received orphan drug designation from the FDA and European Medicines Agency for the treatment of BOS.
  • We believe Fast Track designation provides an opportunity for frequent interactions with the FDA which may potentially expedite the development and registration of LCsAi, said Paola Castellani, CMO at Zambon.
  • Fast Track Designation may allow a therapy to be eligible for several benefits including enhanced interaction with the FDA and the potential for Accelerated Approval and Priority Review at the time of a New Drug Application (NDA) filing, if relevant criteria are met.
  • Additional information about FDA Fast Track designation is available at https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated... .

New 6-year Data for Genentech’s Ocrevus (ocrelizumab) Show Earlier Treatment Initiation Nearly Halves Risk of Needing Walking Aid in Relapsing Multiple Sclerosis

Retrieved on: 
Tuesday, April 28, 2020
Biotechnology, Health, Pharmaceutical, Clinical trials, Managed care, Cytokines, Medical specialties, Monoclonal antibodies, Breakthrough therapy, Genentech, Ocrelizumab, Immunology, Branches of biology, Interferon beta-1a, Interferon, multiple sclerosis, Ocrevus, Genentech in neuroscience, Genentech

These new analyses add additional evidence to the benefit-risk profile of Ocrevus, including the impact of MS on peoples daily lives.

Key Points: 
  • These new analyses add additional evidence to the benefit-risk profile of Ocrevus, including the impact of MS on peoples daily lives.
  • Slowing MS progression earlier in the disease course not just treating relapses may bring additional clinically meaningful outcomes to people living with this disease.
  • People treated with Ocrevus had a 49% reduction in the risk of needing a walking aid compared to those that received interferon beta-1a over 6 years of study (4.3% vs. 7.2%*; p=0.0042).
  • Ocrevus can cause serious side effects, including:
    Infusion reactions: Ocrevus can cause infusion reactions that can be serious and require you to be hospitalized.

Avalyn Pharma Secures $35.5 Million Series B Financing, Broadens Development Portfolio

Retrieved on: 
Monday, April 27, 2020
Biotechnology, Pharmaceutical, Health, Clinical trials, Breakthrough therapy, Chemical compounds, Health, Pulmonology, Idiopathic pulmonary fibrosis, RTT, Nintedanib, Pirfenidone, Pulmonary fibrosis, IPF, Respiratory disease

Avalyn Pharma Inc ., a biopharmaceutical company focused on development of improved therapies for life threatening pulmonary diseases, today announced the completion of a $35.5 million Series B financing.

Key Points: 
  • Avalyn Pharma Inc ., a biopharmaceutical company focused on development of improved therapies for life threatening pulmonary diseases, today announced the completion of a $35.5 million Series B financing.
  • Avalyn has just finished enrollment in a clinical study of two-dose regimens of aerosolized pirfenidone (AP01) in patients with idiopathic pulmonary fibrosis (IPF).
  • Additionally, funds will support a Phase I study of AP02, aerosolized nintedanib, a program entering clinical development after successful preclinical studies in two lung injury models.
  • CLAD occurs despite treatment with maximum systemic doses of multiple anti-rejection drugs and affects the small airways and lung tissue.

NOXXON Presents Latest Clinical Data From the Phase 1/2 NOX-A12 / Keytruda® Combination Trial in Colorectal and Pancreatic Cancer at the AACR Virtual Annual Meeting 2020

Retrieved on: 
Monday, April 27, 2020
Biotechnology, Health, Pharmaceutical, Clinical trials, Oncology, Cancer treatments, Clinical medicine, Medicine, Antineoplastic drugs, Breakthrough therapy, Merck, Pembrolizumab, Noxxon Pharma, Cancer, Pelareorep, Oncolytics Biotech

The safety profile of the combination therapy was consistent with that of pembrolizumab in advanced cancer patients.

Key Points: 
  • The safety profile of the combination therapy was consistent with that of pembrolizumab in advanced cancer patients.
  • Access to AACR Virtual Annual Meeting 2020 is freely available upon registration.
  • Building on extensive clinical experience and safety data, the lead program NOX-A12 has delivered top-line data from a Keytruda combination trial in metastatic colorectal and pancreatic cancer patients and further studies are being planned in these indications.
  • In September 2019 the company initiated an additional trial with NOX-A12 in brain cancer in combination with radiotherapy.

Updated Interim Results from CLASSICAL-Lung, Phase 1b/2 Study of Pepinemab (VX15/2503) in Combination with Avelumab (BAVENCIO®) in Non-Small Cell Lung Cancer Presented at the American Association for Cancer Research (AACR) Virtual Annual Meeting

Retrieved on: 
Monday, April 27, 2020
Health, Merck, Drugs, Avelumab, Breakthrough therapy, Merck Group, Pfizer, Non-small-cell lung carcinoma

The data will be presented in a virtual poster session, available on demand and free to anyone on the AACR website beginning April 27.

Key Points: 
  • The data will be presented in a virtual poster session, available on demand and free to anyone on the AACR website beginning April 27.
  • It is a multi-center, open-label study designed to evaluate the safety and potential efficacy of the combination of pepinemab and avelumab in subjects with advanced NSCLC.
  • Avelumab is co-developed and co-commercialized by Merck KGaA, Darmstadt, Germany and Pfizer Inc.
  • Vaccinex is focused on the development of pepinemab for the treatment of cancer and neurodegenerative diseases including Huntingtons disease.

First Clinical Outcomes Evaluating Six-Week Dosing Schedule for Merck’s KEYTRUDA® (pembrolizumab) Presented at AACR Virtual Annual Meeting I

Retrieved on: 
Monday, April 27, 2020
Research, Clinical trials, Biotechnology, Health, Pharmaceutical, General Health, Other Science, Science, Oncology, Cancer treatments, Clinical medicine, Medicine, Antineoplastic drugs, Breakthrough therapy, Cancer, Melanoma, RTT, Pembrolizumab

Results of the study which represent the first clinical outcomes evaluating Q6W dosing for KEYTRUDA demonstrated efficacy and safety comparable to findings from previous melanoma trials evaluating KEYTRUDA monotherapy.

Key Points: 
  • Results of the study which represent the first clinical outcomes evaluating Q6W dosing for KEYTRUDA demonstrated efficacy and safety comparable to findings from previous melanoma trials evaluating KEYTRUDA monotherapy.
  • In the EU, 400 mg Q6W dosing for KEYTRUDA monotherapy was approved by the European Commission in March 2019.
  • Additionally, PK exposures for KEYTRUDA 400 mg Q6W were within clinical experience with other tested dosing regimens.
  • KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

WPD Pharmaceuticals Announces FDA Application Under The Orphan Drug Act Was Submitted By License Partner for Brain Cancer Drug, Berubicin

Retrieved on: 
Friday, April 24, 2020
Health, Articles, Pharmaceuticals policy, Biotechnology law, Drug discovery, Orphan drug, Orphan Drug Act, Food and Drug Administration, Breakthrough therapy, Food and Drug Administration Safety and Innovation Act

Under a prior developer, Berubicin, then known as RTA 744, was granted ODD by the FDA for the treatment of malignant gliomas.

Key Points: 
  • Under a prior developer, Berubicin, then known as RTA 744, was granted ODD by the FDA for the treatment of malignant gliomas.
  • Additionally, one patient in this study experienced a complete response to his treatment with Berubicin and remains alive today, 14 years after treatment.
  • The Orphan Drug Act ("ODA") provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor.
  • The FDA grants Orphan Drug Designation status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics.

Veracyte and Yale Announce Exclusive License to Advance First Genomic Monitoring Test for Idiopathic Pulmonary Fibrosis

Retrieved on: 
Thursday, April 23, 2020
Health, Genetics, Other Science, Research, Science, Cardiology, Biotechnology, RTT, Idiopathic pulmonary fibrosis, Pulmonary fibrosis, IPF, Organ systems, Monoclonal antibodies, Health, Pneumonia, Breakthrough therapy, Medical specialties, Galecto Biotech, Interstitial lung disease, Health, Genetics, Other Science, Research, Science, Cardiology, Biotechnology, RTT, Idiopathic pulmonary fibrosis, Pulmonary fibrosis, IPF, Organ systems, Monoclonal antibodies, Health, Pneumonia, Breakthrough therapy, Medical specialties, Galecto Biotech, Interstitial lung disease, Envisia, Veracyte, the Yale Office of Cooperative Research

Veracyte, Inc. (Nasdaq: VCYT) and Yale University today announced an exclusive licensing agreement to advance the first genomic test for predicting disease progression in patients with idiopathic pulmonary fibrosis (IPF).

Key Points: 
  • Veracyte, Inc. (Nasdaq: VCYT) and Yale University today announced an exclusive licensing agreement to advance the first genomic test for predicting disease progression in patients with idiopathic pulmonary fibrosis (IPF).
  • The agreement gives Veracyte rights to a 52-gene signature developed by Yale researchers, for use on the nCounter FLEX Analysis System Veracytes exclusively licensed diagnostics platform.
  • Since its founding in 2008, Veracyte has commercialized seven genomic tests and is transforming the diagnosis of thyroid cancer, lung cancer and idiopathic pulmonary fibrosis.
  • Veracyte, Afirma, Percepta, Envisia, Prosigna, LymphMark, and the Veracyte logo are trademarks of Veracyte, Inc. Other trademarks are the property of their respective owners.

Merck Resubmits Supplemental Biologics License Applications (sBLAs) for KEYTRUDA® (pembrolizumab) Six-Week Dosing Schedule

Retrieved on: 
Thursday, April 23, 2020
Oncology, FDA, Health, Clinical trials, Pharmaceutical, Biotechnology, Clinical medicine, Medicine, Cancer treatments, Cancer, Antineoplastic drugs, Breakthrough therapy, Merck, Pembrolizumab, Microsatellite instability, Melanoma, Anaplastic lymphoma kinase

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Key Points: 
  • KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
  • Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
  • The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
  • Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Everest Medicines Announces Approval of Clinical Trial Application by China National Medical Products Administration to Initiate China Registration Study of Sacituzumab Govitecan for the Treatment of Metastatic Triple-Negative Breast Cancer

Retrieved on: 
Wednesday, April 22, 2020
Life sciences, Health, Articles, Breakthrough therapy, Orphan drugs, Sacituzumab govitecan, Pharmacy, Biotechnology

With this CTA, Everest Medicines plans to initiate in the first half of 2020 a pivotal Phase 3 clinical trial of sacituzumab govitecan for the treatment of patients with metastatic triple-negative breast cancer (mTNBC) who have received at least two prior therapies for metastatic disease.

Key Points: 
  • With this CTA, Everest Medicines plans to initiate in the first half of 2020 a pivotal Phase 3 clinical trial of sacituzumab govitecan for the treatment of patients with metastatic triple-negative breast cancer (mTNBC) who have received at least two prior therapies for metastatic disease.
  • The U.S. Food and Drug Administration (FDA) previously granted Fast Track Designation and Breakthrough Therapy Designation to sacituzumab govitecan for the treatment of patients with mTNBC who have received prior therapies for metastatic disease.
  • The FDA is currently reviewing a biologics license application for the therapeutic candidate submitted by Immunomedics for later-line mTNBC.
  • We are pleased to receive CTA approval of sacituzumab govitecan, which represents an opportunity to advance a potential novel treatment option for patients win China.