Orphan drugs

Aldeyra Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for ADX-2191 to Treat Primary Vitreoretinal Lymphoma

Retrieved on: 
Tuesday, July 20, 2021
Oncology, Health, FDA, Surgery, Pharmaceutical, Optical, Biotechnology, Clinical medicine, Medicine, Health care, Orphan drugs, Intravitreal administration, Ophthalmic drug administration, Intravitreal injection, Methotrexate

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company for ADX-2191 (methotrexate for intravitreal injection) for the treatment of primary vitreoretinal lymphoma (PVRL).

Key Points: 
  • Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company for ADX-2191 (methotrexate for intravitreal injection) for the treatment of primary vitreoretinal lymphoma (PVRL).
  • PVRL is an often fatal cancer that creates unique challenges for diagnosis and treatment, stated Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra.
  • The FDAs orphan drug designation fosters an important clinical development and commercialization pathway for ADX-2191, which represents the first methotrexate preparation specifically formulated for intraocular injection.
  • Aldeyra Therapeutics is a biotechnology company developing novel immune-modulating therapies to treat ocular and systemic diseases.

Infinity Pharmaceuticals Hosting Webinar on MARIO-3 and MARIO-275 Phase 2 Trials

Retrieved on: 
Tuesday, July 20, 2021
Biotechnology, Health, Pharmaceutical, Clinical trials, Oncology, Orphan drugs, Drugs, Breast cancer, Health care, Health, Atezolizumab, Breakthrough therapy, Protein-bound paclitaxel, Sarah Cannon Research Institute

The webinar will provide updates on the MARIO-3 TNBC and MARIO-275 UC clinical studies, both of which add eganelisib to approved standard of care regimens.

Key Points: 
  • The webinar will provide updates on the MARIO-3 TNBC and MARIO-275 UC clinical studies, both of which add eganelisib to approved standard of care regimens.
  • MARIO-3 is a Phase 2 triplet combination study evaluating the addition of eganelisib to the approved standard of care combination of Tecentriq and Abraxane in the front-line setting in TNBC.
  • The webinar will feature a presentation by Erika P. Hamilton, M.D.
  • of Sarah Cannon Research Institute at Tennessee Oncolgy, of updated data from MARIO-3 in the context of the current standard of care.

KeifeRx Receives FDA Acceptance of Investigational New Drug for Phase 3 Study of Nilotinib BE in Early Alzheimer's Disease

Retrieved on: 
Tuesday, July 20, 2021
Psychiatry, Cognitive disorders, Branches of biology, Learning disabilities, Psychiatric diagnosis, Neuroscience, Dementia, Orphan drugs, Nilotinib, Alzheimer's disease

The safety and efficacy of a Nilotinib BE will be investigated in individuals with dementia due to Alzheimer's disease with abnormal levels of brain amyloid and supporting clinical diagnosis of early Alzheimer's disease.

Key Points: 
  • The safety and efficacy of a Nilotinib BE will be investigated in individuals with dementia due to Alzheimer's disease with abnormal levels of brain amyloid and supporting clinical diagnosis of early Alzheimer's disease.
  • "Our Phase 3 study provides us the opportunity to gain valuable insights about the potential effectiveness of Nilotinib BE in treating early Alzheimer's.
  • KeifeRx will launch a national, multicenter, placebo-controlled, double-blind study of Nilotinib BE in early Alzheimer's disease.
  • The primary objectives of the study are to investigate the safety and efficacy of Nilotinib BE on the progression of dementia in early Alzheimer's disease.

Iveric Bio Appoints Christopher Simms as Chief Commercial Officer

Retrieved on: 
Monday, July 19, 2021
Biotechnology, Health, Pharmaceutical, Optical, Clinical trials, Life sciences, Health care, Health sciences, Monoclonal antibodies, Orphan drugs, Novartis, Genentech, Ranibizumab, Brolucizumab, Macular degeneration

IVERIC bio, Inc. (Nasdaq: ISEE) announced today the appointment of Christopher Simms to Senior Vice President and Chief Commercial Officer, effective August 2, 2021.

Key Points: 
  • IVERIC bio, Inc. (Nasdaq: ISEE) announced today the appointment of Christopher Simms to Senior Vice President and Chief Commercial Officer, effective August 2, 2021.
  • Mr. Simms joins Iveric Bio from Novartis, where he successfully managed commercial operations for the U.S. Ophthalmics Franchise, launching BEOUV (brolucizumab injection) for wet age-related macular degeneration (AMD).
  • It is exciting to see the advances that Iveric Bio has made, stated Mr. Simms.
  • He joined Novartis in 2017 to build the commercial launch strategy for BEOUV after leading commercial efforts on Lucentis at Genentech.

Kyowa Kirin Announces EU Approval for the Self-administration of CRYSVITA® ▼ (burosumab) to Treat X-Linked Hypophosphataemia (XLH)

Retrieved on: 
Monday, July 19, 2021
Radiology, Pharmaceutical, Genetics, Health, Health, Monoclonal antibodies, Orphan drugs, Breakthrough therapy, Burosumab, X-linked hypophosphatemia, Kyowa Hakko Kirin, Kirin, Health care

The self-administration of CRYSVITA is at the discretion of a treating physician, providing those self-administering are competent and comfortable doing so.

Key Points: 
  • The self-administration of CRYSVITA is at the discretion of a treating physician, providing those self-administering are competent and comfortable doing so.
  • Abdul Mullick, President of Kyowa Kirin International, said: This approval is another significant milestone for the treatment of people living with XLH.
  • Kyowa Kirin is committed to improving the lives of patients and their families by giving healthcare professionals better ways to care for them.
  • By gaining EU approval for the self-administration of CRYSVITA, we have created another valuable option in the care of children, adolescents and adults with XLH.

eFFECTOR Therapeutics Announces Publication of Tomivosertib Preclinical Data in Cell Reports

Retrieved on: 
Friday, July 16, 2021
Cancer treatments, Clinical medicine, Drugs, Breakthrough therapy, Orphan drugs, Cancer immunotherapy, Pembrolizumab, Lung cancer, Effector, Non-small-cell lung carcinoma, ATM serine/threonine kinase

This publication further validates the novel science on which eFFECTORs platform is built, said Steve Worland, Ph.D., president and CEO of eFFECTOR.

Key Points: 
  • This publication further validates the novel science on which eFFECTORs platform is built, said Steve Worland, Ph.D., president and CEO of eFFECTOR.
  • We are proud that our contributions have the potential to broaden the treatment landscape for those impacted by cancer.
  • Tomivosertib is being evaluated in KICKSTART, eFFECTORs randomized, double-blind, placebo-controlled Phase 2b study in non-small cell lung cancer (NSCLC) in combination with pembrolizumab.
  • Neither LWAC nor eFFECTOR gives any assurance that either LWAC or eFFECTOR or the combined company will achieve its expectations.

Bristol Myers Squibb Provides Update on CheckMate -651 Trial Evaluating Opdivo (nivolumab) Plus Yervoy (ipilimumab) Versus EXTREME Regimen as First-Line Treatment for Squamous Cell Carcinoma of the Head and Neck

Retrieved on: 
Friday, July 16, 2021
Stem cells, Biotechnology, Other Health, Health, General Health, Pharmaceutical, Oncology, Other Science, Research, Science, Clinical trials, Drugs, Clinical medicine, Cancer treatments, Orphan drugs, Antineoplastic drugs, Bristol-Myers Squibb, Breakthrough therapy, Cancer immunotherapy, Nivolumab, Ipilimumab, Renal cell carcinoma, Head and neck cancer

Squamous cell carcinoma of the head and neck (SCCHN) accounts for more than 90% of all head and neck cancers.

Key Points: 
  • Squamous cell carcinoma of the head and neck (SCCHN) accounts for more than 90% of all head and neck cancers.
  • OPDIVO (nivolumab), in combination with YERVOY (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
  • OPDIVO (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
  • OPDIVO (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.

Corsair Pharma, Inc. to Present at LifeSci Partners Annual Private Company Summer Symposium

Retrieved on: 
Thursday, July 15, 2021
Health sciences, Pharmacology, Orphan drugs, Prostaglandins, Secondary alcohols, Treprostinil, Health care, Pulmonary hypertension, Corsair, PAH, Transdermal patch

SOUTH SAN FRANCISCO, Calif., July 15, 2021 (GLOBE NEWSWIRE) -- Corsair Pharma, Inc., a private biopharmaceutical company developing a transdermal treprostinil prodrug, today announced it will present at the LifeSci Partners Private Company Summer Symposium, to be held virtually July 21-23, 2021.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., July 15, 2021 (GLOBE NEWSWIRE) -- Corsair Pharma, Inc., a private biopharmaceutical company developing a transdermal treprostinil prodrug, today announced it will present at the LifeSci Partners Private Company Summer Symposium, to be held virtually July 21-23, 2021.
  • Garrett Vygantas, Chief Business Officer of Corsair, will present an update on Corsairs lead product candidate, a prodrug of treprostinil delivered on a proprietary transdermal patch to treat Pulmonary Arterial Hypertension (PAH).
  • Corsair is scheduled to present Wednesday, July 21, 2021, at 2:30 pm Eastern Time.
  • Corsair is also exploring expansion opportunities into other forms of pulmonary hypertension.

Advaxis to Present at the Non-Small Cell Lung Cancer Drug Development Summit

Retrieved on: 
Wednesday, July 14, 2021
Clinical medicine, Cancer treatments, Medicine, Cancer immunotherapy, Lung cancer, Antineoplastic drugs, Breakthrough therapy, Orphan drugs, Advaxis, Pembrolizumab, Merck & Co., Small-cell carcinoma

PRINCETON, N.J., July 14, 2021 (GLOBE NEWSWIRE) -- Advaxis, Inc. (Nasdaq: ADXS), a clinical-stage biotechnology company focused on the development and commercialization of immunotherapy products, today announced that Andres Gutierrez, M.D., Executive Vice President and Chief Medical Officer of Advaxis, will present data from Part B of the Companys Phase 1/2 study of ADXS-503 in combination with Pembrolizumab in patients with metastatic squamous or non-squamous non-small cell lung cancer at the virtual Non-Small Cell Lung Cancer Drug Development Summit on July 15th, 2021.

Key Points: 
  • PRINCETON, N.J., July 14, 2021 (GLOBE NEWSWIRE) -- Advaxis, Inc. (Nasdaq: ADXS), a clinical-stage biotechnology company focused on the development and commercialization of immunotherapy products, today announced that Andres Gutierrez, M.D., Executive Vice President and Chief Medical Officer of Advaxis, will present data from Part B of the Companys Phase 1/2 study of ADXS-503 in combination with Pembrolizumab in patients with metastatic squamous or non-squamous non-small cell lung cancer at the virtual Non-Small Cell Lung Cancer Drug Development Summit on July 15th, 2021.
  • Advaxis, Inc. is a clinical-stage biotechnology company focused on the development and commercialization of proprietary Lm-based antigen delivery products.
  • These immunotherapies are based on a platform technology that utilizes live attenuated Listeria monocytogenes (Lm) bioengineered to secrete antigen/adjuvant fusion proteins.
  • KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.,Kenilworth, N.J., USA.

Foundation Medicine Expands Indication for FoundationOne®Liquid CDx to be Used as a Companion Diagnostic for TABRECTA® (capmatinib)

Retrieved on: 
Friday, July 16, 2021
Medical Supplies, Health, Hospitals, General Health, Pharmaceutical, Biotechnology, Health sciences, Life sciences, Medicine, Orphan drugs, Medical tests, Biostatistics, Companion diagnostic, Medical diagnosis, Novartis, Capmatinib, Foundation Medicine, Medication, FoundationOne Liquid CDx, Foundation Medicine, FOUNDATIONONE LIQUID CDX, FOUNDATION MEDICINE

[2] Today's approval adds to the number of therapies for which both of Foundation Medicines FDA-approved comprehensive genomic tests are listed as companion diagnostics.

Key Points: 
  • [2] Today's approval adds to the number of therapies for which both of Foundation Medicines FDA-approved comprehensive genomic tests are listed as companion diagnostics.
  • FoundationOneCDx, Foundation Medicines tissue test, was approved as a companion diagnostic for TABRECTA in May 2020.
  • TABRECTA is the second therapy for which both of Foundation Medicines FDA-approved tests, FoundationOne CDx and FoundationOne Liquid CDx, are listed as companion diagnostics.
  • Foundation Medicine and FoundationOne are registered trademarks of Foundation Medicine, Inc.
    TABRECTA is a trademark of Novartis.