19 reports

  • 6.1.8 BIOMARIN - TO WHAT EXTENT WILL BIOMARIN CONTINUE TO FOCUS EXCLUSIVELY ON THE TREATMENT OF GENETIC DISORDERS?
  • GENETIC DISORDERS, GLOBAL, KEY MARKETED PRODUCTS AND APPROVED INDICATIONS, 2017

Eliglustat tartrate for the treatment of adults with type ## Gaucher disease.

  • Clinical Trial
  • Genetic Disorder
  • Neurological Disorder
  • Therapy
  • Alexion Pharmaceuticals, Inc.
  • GASTROINTESTINAL DISORDERS, GLOBAL, KEY MARKETED PRODUCTS AND APPROVED INDICATIONS, 2016
  • 4.6 Conclusion

Alexion only markets one product in the gastrointestinal therapy area, namely Soliris.

  • Gastrointestinal Drug
  • Pharmaceutical
  • Therapy
  • Market Size
  • Alexion Pharmaceuticals, Inc.

Therapy areas with one pipeline product such as gastrointestinal and genetic disorders are grouped under the other category.

  • Pharmaceutical
  • Therapy
  • Alexion Pharmaceuticals, Inc.
  • Daiichi Sankyo Company
  • Eisai Co., Ltd.
  • CURAVAC
  • Market trends

Cellular therapy could be one of the specific immunotherapy approaches to treating myasthenia gravis.

  • Neurological Disorder
  • Therapy
  • Alexion Pharmaceuticals, Inc.
  • F. Hoffmann-La Roche Ltd.
  • Flamel Technologies S.A.
  • AUG 20, 2018: FDA ACCEPTS PRIORITY REVIEW OF ALXN1210 AS A TREATMENT FOR PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA IN THE US
  • JUN 19, 2018: ALEXION SUBMITS APPLICATION FOR PRIORITY REVIEW AND APPROVAL OF ALXN1210 AS A TREATMENT FOR PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA IN THE U.S.

At the outset of the ##-week treatment period, patients were randomized in a ##:##:## ratio and received daily SC doses of ##. ## mg/ kg of RA##, ##. ## mg/ kg of RA##, or matching placebo.

  • Autoimmune Disease
  • Monoclonal Antibody
  • Pharmaceutical
  • Therapy
  • Alexion Pharmaceuticals, Inc.
  • NOV 25, 2014: SOLIRIS (ECULIZUMAB) GRANTED ORPHAN DRUG DESIGNATION IN JAPAN FOR THE TREATMENT OF PATIENTS WITH NEUROMYELITIS OPTICA
  • Jul 26, 2019: Alexion receives positive CHMP Opinion for SOLIRIS (Eculizumab) for the treatment Of NMOSD

Among five patients with one or more transfusions in the year prior to the study, only one patient, from Cohort ##, required a transfusion during treatment with ALXN##.

  • Monoclonal Antibody
  • Neurological Disorder
  • Therapy
  • United States
  • Alexion Pharmaceuticals, Inc.
  • JAN 06, 2017: FDA GRANTS ORPHAN DRUG DESIGNATION TO ALXN1210 FOR THE TREATMENT OF PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
  • DEC 14, 2017: WTX101 GRANTED FAST TRACK DESIGNATION BY THE U.S. FDA FOR THE TREATMENT OF WILSON DISEASE

Patients will be randomized in a ##:## ratio to receive treatment with WTX## or SoC.

  • Healthcare
  • Pharmaceutical
  • Therapy
  • Company
  • Alexion Pharmaceuticals, Inc.
  • OCT 23, 2017: FDA APPROVES SOLIRIS (ECULIZUMAB) FOR THE TREATMENT OF PATIENTS WITH GENERALIZED MYASTHENIA GRAVIS
  • AKARI THERAPEUTICS PLC

Among five patients with one or more transfusions in the year prior to the study, only one patient, from Cohort ##, required a transfusion during treatment with ALXN##.

  • Hospital
  • Monoclonal Antibody
  • Pharmaceutical
  • Therapy
  • Alexion Pharmaceuticals, Inc.
  • NOV 25, 2014: SOLIRIS (ECULIZUMAB) GRANTED ORPHAN DRUG DESIGNATION IN JAPAN FOR THE TREATMENT OF PATIENTS WITH NEUROMYELITIS OPTICA
  • APR 15, 2015: CLENE RECEIVES IMPD APPROVAL; PHASE I CLINICAL PROGRAM PLANNED

Average LDH level was ##. ## U/ L at one year of treatment.

  • Monoclonal Antibody
  • Neurological Disorder
  • Pharmaceutical
  • Therapy
  • Alexion Pharmaceuticals, Inc.
  • 18.2 MARKET SHARE ANALYSIS 2014
  • 18. Vendor Landscape

Vidaza is a category ## recommended treatment for patients with intermediate-## and high-risk MDS.

  • Blood Disease
  • Therapy
  • Alexion Pharmaceuticals, Inc.
  • Amgen Inc.
  • Celgene Corporation
  • MARKET SEGMENTATION - BY TOP SELLING DRUGS

TABLE OF CONTENTS ##.

  • Therapy
  • United States
  • Company
  • Market Segment
  • Alexion Pharmaceuticals, Inc.
  • LATEST CLINICAL TRIALS NEWS ON PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
  • CLINICAL TRIAL PROFILE SNAPSHOTS

One patient in the Soliris® arm died from lung cancer (unrelated to Soliris® treatment) during the extension phase of the study.

  • Blood Disease
  • Clinical Trial
  • Medical Biotechnology
  • Therapy
  • Alexion Pharmaceuticals, Inc.
  • Pipeline by Alexion Pharmaceuticals Inc, H2 2016
  • Alkaline Phosphatase (ALKP or Phosphomonoesterase or Glycerophosphatase or Alkaline Phosphohydrolase or EC 3.1.3.1) - Companies Involved in Therapeutics Development

Apart from this, Enobia Pharma has withdrawn one Phase II study of ENB-## for the treatment of Hypophosphatasia.

  • Therapy
  • United States
  • World
  • Product Initiative
  • Alexion Pharmaceuticals, Inc.
  • CLINICAL TRIAL PROFILE SNAPSHOTS

The prominent features of this report are - ##.

  • Blood Disease
  • Clinical Trial
  • Monoclonal Antibody
  • Therapy
  • Alexion Pharmaceuticals, Inc.
  • JUN 01, 2016: EUROPEAN COMMISSION GRANTS ORPHAN DRUG DESIGNATION TO ALXN1210 FOR THE TREATMENT OF PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
  • Complement C5 (C3 And PZP-Like Alpha-2-Macroglobulin Domain-Containing Protein 4) - Featured News & Press Releases

One patient had a human anti-human antibody response, and continued chronic Soliris treatment without apparent adverse effect.

  • Therapy
  • United States
  • World
  • Product Initiative
  • Alexion Pharmaceuticals, Inc.
  • Pipeline by Alexion Pharmaceuticals, Inc., H1 2016
  • Alkaline Phosphatase (ALKP or Phosphomonoesterase or Glycerophosphatase or Alkaline Phosphohydrolase or EC 3.1.3.1) - Drug Profiles

Apart from this, Enobia Pharma has withdrawn one Phase II study of ENB-## for the treatment of Hypophosphatasia.

  • Therapy
  • United States
  • World
  • Product Initiative
  • Alexion Pharmaceuticals, Inc.
  • PARTNERSHIP

FUZEON is a drug indicated for the treatment of Human Immunodeficiency Virus-## Infection.

  • Therapy
  • United States
  • Company
  • Corporate Finance
  • Alexion Pharmaceuticals, Inc.
  • Arterial Calcification - Pipeline by Alexion Pharmaceuticals, Inc., H1 2016
  • Alexion Pharmaceuticals, Inc.

SBC-## is a recombinant enzyme that acts as an enzyme replacement therapy.

  • Therapy
  • United States
  • World
  • Company Operations
  • Alexion Pharmaceuticals, Inc.
  • (ODD) to Soliris (eculizumab) for the prevention of graft rejection following solid organ transplantation.
  • Alexion Pharmaceuticals, Inc. - Recent Pipeline Updates

One patient who received only one ## mg/ kg and one ## mg/ kg dose (instead of ## doses at ## mg/ kg) due to an unrelated SAE was excluded from this analysis, and restarted treatment at week ##.

  • Pharmaceutical
  • Therapy
  • United States
  • Company Operations
  • Alexion Pharmaceuticals, Inc.