Orphan drugs

European Medicines Agency Accepts GBT’s Marketing Authorization Application (MAA) for Oxbryta® (voxelotor) for the Treatment of Hemolytic Anemia in Sickle Cell Disease

Retrieved on: 
Wednesday, January 27, 2021
Clinical medicine, Branches of biology, Hematopathology, Medical specialties, Breakthrough therapy, Orphan drugs, Sickle-cell disease, Anemias, Sickle cell disease, Voxelotor, European Medicines Agency, Book:Hemolytic anemia

GBT is seeking full marketing approval from the EMA for Oxbryta to treat hemolytic anemia in patients with sickle cell disease (SCD) who are 12 years of age and older.

Key Points: 
  • GBT is seeking full marketing approval from the EMA for Oxbryta to treat hemolytic anemia in patients with sickle cell disease (SCD) who are 12 years of age and older.
  • We look forward to working with the EMA to meet our goal of bringing the first treatment for hemolytic anemia in sickle cell disease to European patients as soon as possible.
  • Oxbryta is approved in the United States for the treatment of SCD in adults and children 12 years of age and older.
  • Additionally, Oxbryta has been granted Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), and the European Commission (EC) has designated Oxbryta as an orphan medicinal product for the treatment of patients with SCD.

Sorrento Announces Positive Preliminary Results of Phase 1b Study of COVI-MSC™ for Treatment of ICU COVID-19 Patients

Retrieved on: 
Wednesday, January 27, 2021
Drugs, Monoclonal antibodies, Orphan drugs, Breakthrough therapy, Clinical research

Thisongoingstudy (PSC-CP-004) is a single arm, non-randomized Phase 1b study of the safety and preliminary efficacy ofCOVI-MSCsadministered every other day for three infusions for a total of 1 x 106cells/kg.

Key Points: 
  • Thisongoingstudy (PSC-CP-004) is a single arm, non-randomized Phase 1b study of the safety and preliminary efficacy ofCOVI-MSCsadministered every other day for three infusions for a total of 1 x 106cells/kg.
  • The first three patientsenrolledtolerated treatment well andimproved rapidly.Each of the three patients wasdischarged from the hospital withina weekofstartingthe patientsCOVI-MSC infusions and twopatientswere dischargedonthe dayof theirlast infusion.
  • RTX has completed a phase IB trial for intractable pain associated with cancer and a phase 1B trial in osteoarthritis patients.
  • SEMDEXA is in a pivotal Phase 3 trial for the treatment of lumbosacral radicular pain, or sciatica.ZTlidowas approved by the FDA on February 28, 2018.

Rhythm Pharmaceuticals Announces Positive Data with Setmelanotide in Additional MC4R Pathway Deficiency-related Obesities

Retrieved on: 
Tuesday, January 26, 2021
Health care, Medicine, Branches of biology, Breakthrough therapy, Orphan drugs, Anorectics, Peptides, Setmelanotide, MedWatch, Food and Drug Administration, Pathway, Adverse effect

These new data show that setmelanotide led to meaningful weight loss in approximately 30 percent to greater than 50 percent of treated patients with genetic variants in the MC4R pathway across multiple genes.

Key Points: 
  • These new data show that setmelanotide led to meaningful weight loss in approximately 30 percent to greater than 50 percent of treated patients with genetic variants in the MC4R pathway across multiple genes.
  • Also not included were data from 12 patients who remain ongoing but have not yet reached 12 weeks of therapy.
  • Using this proprietary approach, Rhythm has identified an additional 31 MC4R pathway genes with strong or very strong pathway relevance, which it plans to evaluate in its expanded basket trial.
  • To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch .

European Commission Approves KEYTRUDA® (pembrolizumab) as First-Line Treatment in Adult Patients With Metastatic Microsatellite Instability-High (MSI-H) or Mismatch Repair Deficient (dMMR) Colorectal Cancer

Retrieved on: 
Tuesday, January 26, 2021
Oncology, Health, Clinical trials, General Health, Pharmaceutical, Biotechnology, Cancer treatments, Clinical medicine, Drugs, Breakthrough therapy, Antineoplastic drugs, Orphan drugs, Lung cancer, Bristol-Myers Squibb, Pembrolizumab, Merkel-cell carcinoma, Non-small-cell lung carcinoma, Paclitaxel, Colorectal Cancer in Europe, Merck

Selected KEYTRUDA (pembrolizumab) Indications in the U.S.

Key Points: 
  • Selected KEYTRUDA (pembrolizumab) Indications in the U.S.
    KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
  • KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
  • KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
  • KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC).

RadioMedix & Curium Announce Permanent HCPCS Code for DetectnetTM (copper Cu 64 dotatate injection)

Retrieved on: 
Tuesday, January 26, 2021
Orphan drugs, Radiopharmaceuticals, Branches of biology, Drugs, Medicine, G protein-coupled receptors, Chelating agents, Macrocycles, DOTA-TATE, Somatostatin receptor 2, Copper (64Cu) oxodotreotide, Somatostatin

Ensure safe handling and preparation procedures to protect patients and health care workers from unintentional radiation exposure.

Key Points: 
  • Ensure safe handling and preparation procedures to protect patients and health care workers from unintentional radiation exposure.
  • The uptake of copper Cu 64 dotatate reflects the level of somatostatin receptor density in NETs, however, uptake can also be seen in a variety of other tumors that also express somatostatin receptors.
  • A negative scan after the administration of Detectnet in patients who do not have a history of NET disease does not rule out disease.
  • Non-radioactive somatostatin analogs and copper Cu 64 dotatate competitively bind to somatostatin receptors (SSTR2).

DGAP-News: MorphoSys AG: MorphoSys and I-Mab Announce First Patient Dosed in U.S. Phase 1 Study of MOR210/TJ210 in Patients with Advanced Cancer

Retrieved on: 
Monday, January 25, 2021
Medical specialties, Clinical medicine, C5a, Antibody, MorphoSys, Monoclonal antibodies, Drugs, Orphan drugs, MOR210/TJ210, MorphoSys, I-Mab

Phase 1 Study of MOR210/TJ210 in Patients with Advanced Cancer

Key Points: 
  • Phase 1 Study of MOR210/TJ210 in Patients with Advanced Cancer
    The issuer is solely responsible for the content of this announcement.
  • MOR210/TJ210 is a monoclonal antibody developed by MorphoSys that is directed against complement factor C5a receptor 1 (C5aR1).
  • MOR210/TJ210 demonstrated a good safety profile with no observed adverse effects up to the highest dose tested in non-clinical safety studies.
  • "The data generated from this study will provide valuable information about TJ210/MOR210's safety and tolerability profile and its potential benefits in patients with advanced cancers."

Alector Announces First Participant Dosed in Phase 2 Study Evaluating AL002 in Individuals with Early Alzheimer’s Disease

Retrieved on: 
Monday, January 25, 2021
Monoclonal antibodies, Drugs, TREM2, Dose-ranging study, Orphan drugs

AL002 is a first-in-class TREM2 targeting antibody in Phase 2 clinical development for Alzheimers disease.

Key Points: 
  • AL002 is a first-in-class TREM2 targeting antibody in Phase 2 clinical development for Alzheimers disease.
  • We look forward to efficiently enrolling the Phase 2 study to better understand the potential clinical benefits of AL002.
  • INVOKE-2 is a randomized, double-blind, placebo-controlled, dose-ranging, multi-center Phase 2 study that will enroll approximately 265 participants with early Alzheimers disease at up to 90 sites globally.
  • The primary endpoint of the Phase 2 study is to measure disease progression utilizing the Clinical Dementia Rating Sum Boxes (CDR-SB).

CALQUENCE Met Primary Efficacy Endpoint in Head-to-Head Trial Against ibrutinib in Chronic Lymphocytic Leukemia

Retrieved on: 
Monday, January 25, 2021
Biotechnology, Health, Pharmaceutical, Clinical trials, Oncology, Orphan drugs, Tyrosine kinase inhibitors, Drugs, Health sciences, Health, Antineoplastic drugs, Breakthrough therapy, Acalabrutinib, Ibrutinib, Chronic lymphocytic leukemia, CLL, Clinical endpoint

Positive high-level results from the ELEVATE-RR Phase III trial showed AstraZenecas CALQUENCE (acalabrutinib) met the primary endpoint demonstrating non-inferior progression-free survival (PFS) for adults with previously treated, high-risk chronic lymphocytic leukemia (CLL) compared to ibrutinib.

Key Points: 
  • Positive high-level results from the ELEVATE-RR Phase III trial showed AstraZenecas CALQUENCE (acalabrutinib) met the primary endpoint demonstrating non-inferior progression-free survival (PFS) for adults with previously treated, high-risk chronic lymphocytic leukemia (CLL) compared to ibrutinib.
  • The trial also met a key secondary endpoint for safety, showing patients treated with CALQUENCE had statistically significantly lower incidence of atrial fibrillation compared to patients treated with ibrutinib.
  • Overall, the safety and tolerability of CALQUENCE were consistent with the profile seen in the broader CALQUENCE clinical development program.
  • Prevalence and Economic Burden of Chronic Lymphocytic Leukemia (CLL) in the Era of Oral Targeted Therapies.

U.S. Food and Drug Administration Approves OPDIVO® (nivolumab) in Combination with CABOMETYX® (cabozantinib) as First-line Treatment for Patients with Advanced Renal Cell Carcinoma

Retrieved on: 
Friday, January 22, 2021
Oncology, FDA, Health, Clinical trials, Pharmaceutical, Biotechnology, Clinical medicine, Drugs, Chemical compounds, Antineoplastic drugs, Bristol-Myers Squibb, Breakthrough therapy, Orphan drugs, Monoclonal antibodies, Nivolumab, Cabozantinib, Ipilimumab, Renal cell carcinoma

Withhold or permanently discontinue OPDIVO and YERVOY depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information).

Key Points: 
  • Withhold or permanently discontinue OPDIVO and YERVOY depending on severity (please see section 2 Dosage and Administration in the accompanying Full Prescribing Information).
  • Consider administration of other systemic immunosuppressants in patients whose immune-mediated adverse reactions are not controlled with corticosteroid therapy.
  • In patients receiving OPDIVO and cabozantinib, Grades 3 and 4 increased ALT or AST were seen in 11% of patients.
  • Nivolumab plus cabozantinib versus sunitinib in first-line treatment for advanced renal cell carcinoma: first results from the randomized phase 3 CheckMate 9ER trial.

Exelixis Announces U.S. FDA Approval of CABOMETYX® (cabozantinib) in Combination with OPDIVO® (nivolumab) as a First-Line Treatment for Patients with Advanced Renal Cell Carcinoma

Retrieved on: 
Friday, January 22, 2021
Biotechnology, Health, Pharmaceutical, Clinical trials, Oncology, Chemical compounds, Drugs, Cancer treatments, Antineoplastic drugs, Orphan drugs, Breakthrough therapy, Bristol-Myers Squibb, Cabozantinib, Nivolumab, Sunitinib, Renal cell carcinoma, Exelixis, CheckMate -9ER, RCC, CABOMETYX® (cabozantinib), Exelixis

Exelixis, Inc. (NASDAQ: EXEL) today announced that the U.S. Food and Drug Administration (FDA) approved CABOMETYX (cabozantinib) for patients with advanced renal cell carcinoma (RCC) as a first-line treatment in combination with OPDIVO (nivolumab).

Key Points: 
  • Exelixis, Inc. (NASDAQ: EXEL) today announced that the U.S. Food and Drug Administration (FDA) approved CABOMETYX (cabozantinib) for patients with advanced renal cell carcinoma (RCC) as a first-line treatment in combination with OPDIVO (nivolumab).
  • The findings for the combination of CABOMETYX and OPDIVO in the CheckMate -9ER trial make the FDA approval of this combination a notable development for the patient community.
  • CheckMate -9ER is an open-label, randomized (1:1), multi-national phase 3 trial evaluating patients with previously untreated advanced or metastatic renal cell carcinoma with a clear cell component.
  • The primary efficacy analysis compares the doublet combination regimen of CABOMETYX and OPDIVO versus sunitinib in all randomized patients.