Orphan drugs

KEYTRUDA® (pembrolizumab) Plus Chemotherapy Before Surgery and Continued as a Single Agent After Surgery Showed Statistically Significant Event-Free Survival (EFS) Result Versus Neoadjuvant Chemotherapy Alone in High-Risk Early-Stage TNBC

Retrieved on: 
Thursday, July 15, 2021
Other Health, Research, General Health, Pharmaceutical, Oncology, Infectious diseases, Clinical trials, Science, Biotechnology, FDA, Other Science, Health, Clinical medicine, Health sciences, Cancer treatments, Orphan drugs, Breakthrough therapy, Antineoplastic drugs, Cancer immunotherapy, Pembrolizumab, Adjuvant therapy, Neoadjuvant therapy, Therapy, Programmed cell death protein 1, Triple-Negative Breast Cancer (TNBC), Merck, TRIPLE-NEGATIVE BREAST CANCER (TNBC), MERCK

This is the first time an anti-PD-1/L1 therapy has demonstrated a statistically significant EFS result as combined neoadjuvant and adjuvant therapy for these patients.

Key Points: 
  • This is the first time an anti-PD-1/L1 therapy has demonstrated a statistically significant EFS result as combined neoadjuvant and adjuvant therapy for these patients.
  • KEYNOTE-522 was designed to study whether the combined neoadjuvant and adjuvant regimen with KEYTRUDA could help treat the cancer earlier.
  • Immune-mediated AEs led to death in 0.3% of patients receiving the KEYTRUDA regimen (n=2) and no patients receiving the chemotherapy-placebo regimen.
  • KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.

City of Hope and Osel Announce Exclusive License for Intellectual Property on the Use of Live Biotherapeutic Product CBM588 for Oncology Therapeutic Applications

Retrieved on: 
Wednesday, July 14, 2021
Oncology, Health, Clinical trials, Research, Science, Pharmaceutical, Biotechnology, Drugs, Cancer treatments, Clinical medicine, Bristol-Myers Squibb, Breakthrough therapy, Orphan drugs, Antineoplastic drugs, Ipilimumab, Nivolumab, Renal cell carcinoma, Pembrolizumab, Atezolizumab, Osel Inc., City of Hope, Miyarisan, OSEL INC., CITY OF HOPE, MIYARISAN

The experimental treatment was evaluated in a City of Hope investigator-initiated Phase 1b trial.

Key Points: 
  • The experimental treatment was evaluated in a City of Hope investigator-initiated Phase 1b trial.
  • 4513 titled First results of a randomized phase IB study comparing nivolumab/ipilimumab with or without CBM588 in patients with metastatic renal cell carcinoma is available here .
  • We look forward to working with City of Hope to further investigate how CBM588 can improve the lives of cancer patients.
  • Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy .

New TAVALISSE® Data Analyses To Be Presented at International Society on Thrombosis and Haemostasis (ISTH) 2021 Congress

Retrieved on: 
Wednesday, July 14, 2021
Blood, Hematology, Health, Drugs, Orphan drugs, Coagulation system, Fostamatinib, Organophosphates, Prodrugs, Hemostasis, Thrombosis, Thrombocythemia

All ePosters will be made available online during the Poster Networking Session on the conference website on Saturday, July 17, 2021, at 4:00 p.m. Eastern Time.

Key Points: 
  • All ePosters will be made available online during the Poster Networking Session on the conference website on Saturday, July 17, 2021, at 4:00 p.m. Eastern Time.
  • Incidence of bleeding events and use of rescue therapy continually decreased in these 58 patients over the first year of treatment with fostamatinib.
  • Findings include:
    A reduced need for office visits due to oral administration, easy titration, and low incidence of thrombocytosis.
  • Fostamatinib's unique mechanism of action provide increased hemostasis and may reduce the risk of thrombosis.

FDA Grants Alkeus Pharmaceuticals Breakthrough Therapy Designation for ALK-001 (C20-D3-vitamin A) for the Treatment of Stargardt Disease

Retrieved on: 
Wednesday, July 14, 2021
Health sciences, Breakthrough therapy, Health, Pharmacy, Stargardt disease, Orphan drugs

SOMERVILLE, Mass., July 14, 2021 (GLOBE NEWSWIRE) -- Alkeus Pharmaceuticals, Inc., a private, late-stage biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to ALK-001 for the treatment of Stargardt Disease.

Key Points: 
  • SOMERVILLE, Mass., July 14, 2021 (GLOBE NEWSWIRE) -- Alkeus Pharmaceuticals, Inc., a private, late-stage biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to ALK-001 for the treatment of Stargardt Disease.
  • ALK-001 is the only drug to receive Breakthrough Therapy Designation for Stargardt Disease.
  • Obtaining Breakthrough Therapy Designation is a transformative milestone in Stargardt disease, said Leonide Saad, PhD, CEO of Alkeus Pharmaceuticals.
  • The FDA granted ALK-001 Breakthrough Therapy Designation after reviewing data from Alkeus Pharmaceuticals Phase 2, double-masked, randomized, placebo-controlled trial in Stargardt disease.

TFF Pharmaceuticals Announces Completion of Enrollment and Preliminary Data from its Phase 1 Clinical Trial of Tacrolimus Inhalation Powder

Retrieved on: 
Tuesday, July 13, 2021
Branches of biology, Drugs, Biology, Orphan drugs, Astellas Pharma, Immunosuppressants, Macrolides, Tacrolimus, Magnesium in biology, Dose

In the Single Ascending Dose (SAD) phase of the trial, single doses of inhaled tacrolimus of 0.5 mg, 1.0 mg, 2.5 mg to 5.0 mg were administered to healthy subjects.

Key Points: 
  • In the Single Ascending Dose (SAD) phase of the trial, single doses of inhaled tacrolimus of 0.5 mg, 1.0 mg, 2.5 mg to 5.0 mg were administered to healthy subjects.
  • Peak blood levels were measured 15 min after dosing, and trough blood levels were measured 12 hours after dosing for each subject.
  • These data demonstrate that low dosing of Tacrolimus Inhalation Powder (0.5-1.0 mg) can achieve blood levels that are sufficient for efficacious immunosuppression.
  • TFF Pharmaceuticals has two lead drug candidates: Voriconazole Inhalation Powder and Tac-Lac Inhalation Powder.

Galecto Resumes Recruitment in Phase 2b Global GALACTIC-1 Trial of GB0139 for Idiopathic Pulmonary Fibrosis

Retrieved on: 
Tuesday, July 13, 2021
Idiopathic pulmonary fibrosis, Pulmonary fibrosis, ClinicalTrials.gov, Orphan drugs, Health, Medicine

Following the recommendation from an independent Data Safety Monitoring Board in March 2021 to modify the GALACTIC-1 trial protocol, 38 patients continued treatment in this Phase 2b trial.

Key Points: 
  • Following the recommendation from an independent Data Safety Monitoring Board in March 2021 to modify the GALACTIC-1 trial protocol, 38 patients continued treatment in this Phase 2b trial.
  • Galecto anticipates that topline data from the GALACTIC-1 trial will be available by mid-2023.
  • We are pleased to continue our Phase 2b GALACTIC-1 trial of GB0139 in IPF patients.
  • The compound is not taken up via the oral route and is being developed as an inhaled therapeutic in Idiopathic Pulmonary Fibrosis (https://clinicaltrials.gov/ct2/show/NCT03832946).

Idiopathic Pulmonary Fibrosis (IPF) Clinical Landscape Market Report 2021 - ResearchAndMarkets.com

Retrieved on: 
Monday, July 12, 2021
Pharmaceutical, Health, Clinical trials, Medical devices, Idiopathic pulmonary fibrosis, Lung disorders, Boehringer Ingelheim, Pulmonary fibrosis, IPF, Health, Orphan drugs, Organ systems, Monoclonal antibodies, Pneumonia, Fresolimumab

The "Market Spotlight: Idiopathic pulmonary fibrosis (IPF)" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Market Spotlight: Idiopathic pulmonary fibrosis (IPF)" report has been added to ResearchAndMarkets.com's offering.
  • This Market Spotlight report covers the Idiopathic Pulmonary Fibrosis (IPF) market, comprising key marketed and pipeline drugs, clinical trials, recent events and analyst opinion, upcoming events, probability of success, 10-year disease incidence and prevalence forecasts, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.
  • The majority of industry-sponsored drugs in active clinical development for IPF are in Phase I.
  • Roche leads the industry sponsors with the highest overall number of clinical trials for IPF, closely followed by Boehringer Ingelheim.

Halozyme Announces Janssen Receives U.S. FDA Approval of DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) in Combination with Pomalidomide and Dexamethasone for Patients with Multiple Myeloma After First or Subsequent Relapse

Retrieved on: 
Monday, July 12, 2021
Drugs, Combination drugs, Hyaluronidase, Breakthrough therapy, Orphan drugs, Daratumumab/hyaluronidase, Halozyme, Daratumumab, Multiple myeloma

The approval marks Janssen's sixth indication for DARZALEX FASPRO in the treatment of multiple myeloma.

Key Points: 
  • The approval marks Janssen's sixth indication for DARZALEX FASPRO in the treatment of multiple myeloma.
  • "This introduces a new delivery option for multiple myeloma patients in the U.S. being treated with this regimen."
  • DARZALEX FASPRO is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE drug delivery technology.
  • Except as required by law, Halozyme undertakes no duty to update forward-looking statements to reflect events after the date of this release.

Janssen Announces U.S. FDA Approval of DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) in Combination with Pomalidomide and Dexamethasone for Patients with Multiple Myeloma After First or Subsequent Relapse

Retrieved on: 
Monday, July 12, 2021
Drugs, Health care, Orphan drugs, Medicine, Daratumumab, Multiple myeloma, CD38, Pomalidomide, Monoclonal antibody, AL amyloidosis, Amyloidosis, Isatuximab

The approval follows the regulatory submission to the FDA in November 2020 and marks the sixth indication for DARZALEX FASPROin the treatment of multiple myeloma.

Key Points: 
  • The approval follows the regulatory submission to the FDA in November 2020 and marks the sixth indication for DARZALEX FASPROin the treatment of multiple myeloma.
  • "Today's approval further distinguishes DARZALEX FASPRO in the treatment of multiple myeloma as the first and only subcutaneously administered anti-CD38 monoclonal antibody approved in combination with the widely used pomalidomide and dexamethasone regimen."
  • DARZALEX FASPRO is the only CD38-directed antibody approved to be given subcutaneously to treat patients with multiple myeloma and now AL amyloidosis.
  • Both systemic administration-related reactions, including severe or life-threatening reactions, and local injection-site reactions can occur with DARZALEX FASPRO.

KalVista Pharmaceuticals Presents Data Showing Single On-Demand Treatment with Orally Administered KVD900 Significantly Slows Progression and Accelerates Resolution of Attacks in Patients with HAE

Retrieved on: 
Monday, July 12, 2021
Biotechnology, Pharmaceutical, Health, Clinical trials, Complement deficiency, Organ systems, Hereditary angioedema, Serpinopathies, Medical specialties, Angioedema, Hae, Clinical medicine, Orphan drugs

As KVD900 halted attack progression and resolved attacks more quickly in patients with HAE, while demonstrating a good safety and tolerability profile, it could be a valued choice for physicians and patients in managing HAE.

Key Points: 
  • As KVD900 halted attack progression and resolved attacks more quickly in patients with HAE, while demonstrating a good safety and tolerability profile, it could be a valued choice for physicians and patients in managing HAE.
  • The late-breaking poster, titled A single on-demand treatment with orally administered KVD900 significantly slows progression and accelerates resolution of attacks in patients with hereditary angioedema (HAE): results of a phase 2, placebo-controlled, double-blind cross-over trial, contains the comprehensive data set from the companys Phase 2 clinical trial of KVD900 in HAE patients.
  • KalVista presented four other posters at EAACI related to the HAE clinical landscape and unmet needs, as well as preclinical data from other oral molecules.
  • Patients with nC1-HAE may represent a more sizeable population of patients with HAE in the U.S. than previously suspected.