Orphan drugs

PharmAbcine signs a research collaboration agreement with Wuxi Shuangliang Biotechnology (SLBio) to evaluate a combination therapy to treat NSCLC

Monday, August 3, 2020 - 2:00pm

Olinvacimab, the leading clinical candidate being developed by PharmAbcine, has shown impressive safety profile and efficacy in clinical studies.

Key Points: 
  • Olinvacimab, the leading clinical candidate being developed by PharmAbcine, has shown impressive safety profile and efficacy in clinical studies.
  • It is currently in a phase II clinical trial in Australia and USA in bevacizumab non responding rGBM patients.
  • In addition, two phase Ib combination trials of olinvacimab and pembrolizumab are ongoing in mTNBC and in rGBM patients.
  • Thisstudy will give us a tremendous opportunity to explore an optimal combination of angiogenesis regulator and 3rd generation EGFR inhibitor to treat NSCLC."

Active Biotech announces first patient dosed in phase 1b/2a study of tasquinimod use in treatment of multiple myeloma

Monday, August 3, 2020 - 7:30am

Tasquinimod is developed as a new immunomodulatory treatment for multiple myeloma.

Key Points: 
  • Tasquinimod is developed as a new immunomodulatory treatment for multiple myeloma.
  • Tasquinimod has previously been studied as an anti-cancer agent in patients with solid cancers, including a phase 3 randomized trial in patients with metastatic prostate cancer.
  • Tasquinimod has demonstrated a clear therapeutic potentialin preclinical models of multiple myeloma, when used as a single agent and in combination with standard multiple myeloma therapy.
  • Tasquinimod, an oral immunomodulator, is in clinical development for treatment of multiple myeloma, NCT04405167.

DGAP-News: FDA Approves Monjuvi(R) (tafasitamab-cxix) in Combination with Lenalidomide for the Treatment of Adult Patients with Relapsed or Refractory Diffuse Large B-cell Lymphoma (DLBCL)

Saturday, August 1, 2020 - 1:00am

The FDA decision represents the first approval of a second-line treatment for adult patients who progressed during or after first-line therapy.

Key Points: 
  • The FDA decision represents the first approval of a second-line treatment for adult patients who progressed during or after first-line therapy.
  • "The FDA approval of Monjuvi in combination with lenalidomide helps address an urgent unmet medical need for patients with relapsed or refractory DLBCL in the United States," said Herv Hoppenot, Chief Executive Officer, Incyte.
  • "Today's FDA decision offers new hope for patients with this aggressive form of DLBCL who progressed during or after first-line therapy."
  • The FDA previously granted Fast Track and Breakthrough Therapy Designation for the combination of Monjuvi and lenalidomide in relapsed or refractory DLBCL.

FDA Approves Monjuvi® (tafasitamab-cxix) in Combination With Lenalidomide for the Treatment of Adult Patients With Relapsed or Refractory Diffuse Large B-cell Lymphoma (DLBCL)

Saturday, August 1, 2020 - 12:51am

The FDA decision represents the first approval of a second-line treatment for adult patients who progressed during or after first-line therapy.

Key Points: 
  • The FDA decision represents the first approval of a second-line treatment for adult patients who progressed during or after first-line therapy.
  • The FDA approval of Monjuvi in combination with lenalidomide helps address an urgent unmet medical need for patients with relapsed or refractory DLBCL in the United States, said Herv Hoppenot, Chief Executive Officer, Incyte.
  • The FDA approval was based on data from the MorphoSys-sponsored Phase 2 L-MIND study, an open label, multicenter, single arm trial of Monjuvi in combination with lenalidomide as a treatment for adult patients with relapsed or refractory DLBCL.
  • The FDA previously granted Fast Track and Breakthrough Therapy Designation for the combination of Monjuvi and lenalidomide in relapsed or refractory DLBCL.

Genmab Announces European Myeloma Network and Janssen Achieve Positive Topline Results from Phase 3 APOLLO Study of Daratumumab in Combination with Pomalidomide and Dexamethasone in Relapsed or Refractory Multiple Myeloma

Friday, July 31, 2020 - 8:38pm

We are pleased with these positive results for daratumumab, administered as a subcutaneous formulation, in combination with pomalidomide and dexamethasone.

Key Points: 
  • We are pleased with these positive results for daratumumab, administered as a subcutaneous formulation, in combination with pomalidomide and dexamethasone.
  • This Phase 3 (NCT03180736), randomized, open-label, multicenter study included 304 patients with multiple myeloma who have previously been treated with lenalidomide and a PI.
  • In the original design of the study, patients in the daratumumab plus Pd arm were treated with the IV formulation of daratumumab.
  • A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and refractory and frontline multiple myeloma settings.

Eisai: Application for Additional Indication of Anti Cancer Agent Lenvima for Unresectable Thymic Carcinoma Submitted in Japan

Friday, July 31, 2020 - 1:31am

a subsidiary of Merck & Co., Inc., Kenilworth, N.J., U.S.A., announced today that Eisai has submitted an application in Japan for the additional indication of treatment of unresectable thymic carcinoma for multiple receptor tyrosine kinase inhibitor LENVIMA (generic name: lenvatinib mesylate).

Key Points: 
  • a subsidiary of Merck & Co., Inc., Kenilworth, N.J., U.S.A., announced today that Eisai has submitted an application in Japan for the additional indication of treatment of unresectable thymic carcinoma for multiple receptor tyrosine kinase inhibitor LENVIMA (generic name: lenvatinib mesylate).
  • In June 2020, LENVIMA received orphan drug designation in Japan for unresectable thymic carcinoma.
  • This application is based on the results of an open-label, single-arm, multicenter, investigator-initiated clinical phase II study (NCCH1508) conducted in Japan, evaluating LENVIMA as a single agent in 42 patients with thymic carcinoma previously treated with at least one platinum-based regimen.
  • This study met its endpoint as the lower value of the CI exceeded the pre-specified statistical criteria, a threshold ORR of 10%.

Imara Announces IMR-687 Granted Fast Track Designation and Rare Pediatric Disease Designation for Treatment of Beta-Thalassemia

Thursday, July 30, 2020 - 12:00pm

The FDA previously granted Orphan Drug designation for IMR-687 for the treatment of patients with beta-thalassemia and Orphan Drug, Fast Track and Rare Pediatric Disease designations for the treatment of patients with sickle cell disease.

Key Points: 
  • The FDA previously granted Orphan Drug designation for IMR-687 for the treatment of patients with beta-thalassemia and Orphan Drug, Fast Track and Rare Pediatric Disease designations for the treatment of patients with sickle cell disease.
  • We are pleased to receive from the FDA both Fast Track designation and Rare Pediatric Disease designation for IMR-687.
  • Fast Track designation helps us create the opportunity to potentially accelerate the development of IMR-687 in beta-thalassemia.
  • Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process.

Bristol Myers Squibb and bluebird bio Announce Submission of Biologics License Application (BLA) to FDA for Idecabtagene Vicleucel (Ide-cel, bb2121) for Adults with Relapsed and Refractory Multiple Myeloma

Wednesday, July 29, 2020 - 9:16pm

The company is committed to working with FDA to progress both applications and achieve the remaining regulatory milestones required by the CVR.

Key Points: 
  • The company is committed to working with FDA to progress both applications and achieve the remaining regulatory milestones required by the CVR.
  • Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between Bristol Myers Squibb and bluebird bio.
  • Idecabtagene vicleucel (ide-cel; bb2121), a BCMA-targeted CAR T cell therapy, in patients with relapsed and refractory multiple myeloma (RRMM): initial KarMMa results.
  • KarMMa-RW: a study of real world treatment patterns in heavily pretreated patients with relapsed and refractory multiple myeloma (RRMM) and comparison of outcomes to KarMMa.

AMRYT TO HOST KEY OPINION LEADER CALL ON EPIDERMOLYSIS BULLOSA DISEASE LANDSCAPE

Wednesday, July 29, 2020 - 12:00pm

Amryt is a biopharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare and orphan diseases.

Key Points: 
  • Amryt is a biopharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare and orphan diseases.
  • Amryt comprises a strong and growing portfolio of commercial and development assets.
  • FILSUVEZ has been granted Rare Pediatric Disease Designation and has also received a Fast Track Designation from the FDA.
  • Such forward-looking statements reflect the Companys current beliefs and assumptions and are based on information currently available to management.

Larimar Therapeutics Announces Positive Opinion on Orphan Drug Designation Received from the European Medicines Agency for CTI-1601 for the Treatment of Friedreich’s Ataxia

Tuesday, July 28, 2020 - 1:00pm

The U.S. Food and Drug Administration (FDA) previously granted Orphan Drug, Fast Track and Rare Pediatric Disease designations to CTI-1601 for the treatment of FA.

Key Points: 
  • The U.S. Food and Drug Administration (FDA) previously granted Orphan Drug, Fast Track and Rare Pediatric Disease designations to CTI-1601 for the treatment of FA.
  • Larimar expects that the European Commission, based on this positive opinion of the COMP, will formally grant the orphan drug designation for the European Union (EU) this year.
  • Phase 1 trial of CTI-1601, which has the potential to become the first frataxin replacement therapy for patients with FA.
  • Orphan drug designation in the EU is granted by the European Commission based on a positive opinion issued by the EMA COMP.