Orphan drugs

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

Friday, January 22, 2021 - 6:18pm

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

Friday, January 22, 2021 - 6:10pm

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.

FDA Approves First Extended-Release, Injectable Drug Regimen for Adults Living with HIV

Thursday, January 21, 2021 - 9:21pm

"Currently, the standard of care for patients with HIV includes patients taking daily pills to adequately manage their condition.

Key Points: 
  • "Currently, the standard of care for patients with HIV includes patients taking daily pills to adequately manage their condition.
  • "Having this treatment available for some patients provides an alternative for managing this chronic condition."
  • The most common adverse reactions with Cabenuva were injection site reactions, fever (pyrexia), fatigue, headache, musculoskeletal pain, nausea, sleep disorders, dizziness and rash.
  • Cabenuva and Vocabria were granted Fast Track and Priority Review designation by the FDA.

Incyte Announces Acceptance and Priority Review of BLA for Retifanlimab as a Potential Treatment for Patients with Squamous Cell Carcinoma of the Anal Canal (SCAC)

Thursday, January 21, 2021 - 9:30pm

Retifanlimab has been granted Orphan Drug Designation by the FDA for the treatment of anal cancer, along with Priority Review.

Key Points: 
  • Retifanlimab has been granted Orphan Drug Designation by the FDA for the treatment of anal cancer, along with Priority Review.
  • The FDA grants Priority Review to medicines that may offer a major advance in treatment where none currently exists.
  • Retifanlimab has been granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of anal cancer.
  • The role of systemic chemotherapy and multidisciplinary management in improving the overall survival of patients with metastatic squamous cell carcinoma of the anal canal.

First Patients Tested with InVisionFirst®-Lung in EORTC Sponsored Phase II NSCLC Study

Wednesday, January 20, 2021 - 12:00pm

The aim of this academic trial, announced in 2019 and being supported by Pfizer, is to examine the resistance to lorlatinib in ALK positive NSCLC patients.

Key Points: 
  • The aim of this academic trial, announced in 2019 and being supported by Pfizer, is to examine the resistance to lorlatinib in ALK positive NSCLC patients.
  • While in the past these patients were known to respond well to ALK inhibition therapies, such as lorlatinib, drug resistance in patients has been increasing.
  • A total of 100 patients will be involved in two study designs run in parallel across 30 participating EORTC sites across Europe.
  • The Phase II study is a single arm, multicenter study.

Doptelet® (avatrombopag) approved in the EU for treatment of ITP

Wednesday, January 20, 2021 - 8:01am

Immune thrombocytopenia (ITP) is an autoimmune disorder characterised by low numbers of platelets, leading to bruising and an increased risk of bleeding.

Key Points: 
  • Immune thrombocytopenia (ITP) is an autoimmune disorder characterised by low numbers of platelets, leading to bruising and an increased risk of bleeding.
  • "The European approval of Doptelet for the ITP indication is a milestone for people living with ITP across Europe.
  • In addition to current indications, the new indication reads;
    Doptelet is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments (e.g.
  • In June 2019, Doptelet was approved by the FDA for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Doptelet® (avatrombopag) approved in the EU for treatment of ITP

Wednesday, January 20, 2021 - 7:58am

Immune thrombocytopenia (ITP) is an autoimmune disorder characterised by low numbers of platelets, leading to bruising and an increased risk of bleeding.

Key Points: 
  • Immune thrombocytopenia (ITP) is an autoimmune disorder characterised by low numbers of platelets, leading to bruising and an increased risk of bleeding.
  • "The European approval of Doptelet for the ITP indication is a milestone for people living with ITP across Europe.
  • In addition to current indications, the new indication reads;
    Doptelet is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments (e.g.
  • In June 2019, Doptelet was approved by the FDA for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Major shareholder announcement

Monday, January 18, 2021 - 3:08pm

Danske Bank A/S shareholding consists of a 2.24% indirect and 1.25% direct ownership through Danica Pension Livsforsikringsaktieselskab, Danica Pension Frskringsaktiebolag, Investeringsforeningen Danske Invest and Danske Invest SICAV.

Key Points: 
  • Danske Bank A/S shareholding consists of a 2.24% indirect and 1.25% direct ownership through Danica Pension Livsforsikringsaktieselskab, Danica Pension Frskringsaktiebolag, Investeringsforeningen Danske Invest and Danske Invest SICAV.
  • The Company is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases.
  • Arimoclomol has received Fast Track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC, IBM, and ALS.
  • In addition, arimoclomol has received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC.

Immunotherapy Veteran Robert Knight, MD, Joins NexImmune as Chief Medical Officer

Monday, January 18, 2021 - 1:30pm

Dr. Knight is a seasoned executive with extensive experience leading early- and late-stage immunotherapy product development teams.

Key Points: 
  • Dr. Knight is a seasoned executive with extensive experience leading early- and late-stage immunotherapy product development teams.
  • During his tenure at Celgene, he helped lead the development of the companys IMiD and targeted therapy programs, including thalidomide, lenalidomide, and enasidenib.
  • We are excited to welcome Bob to the NexImmune team, said Scott Carmer, NexImmune Chief Executive Officer.
  • I am thrilled to join the NexImmune team as Chief Medical Officer at this important point in the Companys lifecycle, said Dr. Knight.

SinoMab Dosed First Healthy Subject in Phase I Clinical Trial of SN1011 in China

Sunday, January 17, 2021 - 11:30pm

Medical staff from Shuguan Hospital in Shanghai conducted the Phase I clinical trial of SN1011.

Key Points: 
  • Medical staff from Shuguan Hospital in Shanghai conducted the Phase I clinical trial of SN1011.
  • SN1011 differentiates from existing BTK inhibitors currently available in the market, such as Ibrutinib, in terms of selectivity and affinity.
  • The Phase I clinical trial was conducted in Shuguan Hospital in Shanghai, aiming to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics and recommended Phase II dose of SN1011 in treatment for autoimmune diseases.
  • Dr. Shui On LEUNG, Chairman, Executive Director and Chief Executive Officer of SinoMab, said, "SN1011 successfully completed its first dose, representing another key R&D asset entering the stage of clinical trial following our flagship product, SM03.