Marketing authorization

Orphazyme initiates rolling submission of New Drug Application for arimoclomol with US FDA in Niemann-Pick disease Type C

Friday, May 29, 2020 - 6:12am

Arimoclomol has received FDA Fast Track and Breakthrough Therapy Designations for the treatment of NPC, in addition to Orphan Drug and Rare Pediatric Disease Designations.

Key Points: 
  • Arimoclomol has received FDA Fast Track and Breakthrough Therapy Designations for the treatment of NPC, in addition to Orphan Drug and Rare Pediatric Disease Designations.
  • The FDAs rolling review allows the company to submit portions of the NDA to the FDA as they are completed.
  • Orphazyme expects to complete submission of the remaining portions of the NDA to the FDA in the next couple of months.
  • Orphazyme expects to submit a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for arimoclomol in NPC in H2 2020.

Alnylam Announces U.S. Food and Drug Administration Has Granted Priority Review of the Lumasiran New Drug Application for the Treatment of Primary Hyperoxaluria Type 1

Tuesday, May 26, 2020 - 12:00pm

Award of Priority Review status adds to the previous grants of Breakthrough Therapy and Pediatric Rare Disease Designations.

Key Points: 
  • Award of Priority Review status adds to the previous grants of Breakthrough Therapy and Pediatric Rare Disease Designations.
  • In addition, the Marketing Authorisation Application (MAA) for lumasiran has been submitted to and validated by the European Medicines Agency (EMA).
  • Lumasiran has also previously received Orphan Drug Designations for the treatment of PH1 in the U.S. and Europe and has received a Priority Medicines (PRIME) designation from the EMA.
  • Lumasiran is an investigational, subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of primary hyperoxaluria type 1 (PH1).

Karyopharm Announces Investor Conference Call to Discuss Phase 3 BOSTON Data To Be Presented at the American Society of Clinical Oncology 2020 Virtual Scientific Program

Tuesday, May 26, 2020 - 12:00pm

The conference call will follow the presentation of this data at the upcoming American Society of Clinical Oncology (ASCO) 2020 Virtual Scientific Program.

Key Points: 
  • The conference call will follow the presentation of this data at the upcoming American Society of Clinical Oncology (ASCO) 2020 Virtual Scientific Program.
  • The investor conference call is scheduled for Friday, May 29, 2020 at 1:00 p.m.
  • Karyopharm has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) with a request for conditional approval of selinexor.
  • In May 2020, Karyopharm submitted a supplemental New Drug Application based on data from the Phase 3 BOSTON study.

QPPV Online Training - Technical Training Program To Become A Qualified Pharmacovigilance Professional

Thursday, May 7, 2020 - 5:45pm

DUBLIN, May 7, 2020 /PRNewswire/ -- The "QPPV Online Training - A Technical Training Program To Be Qualified Pharmacovigilance Professional (QPPV)" training has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • DUBLIN, May 7, 2020 /PRNewswire/ -- The "QPPV Online Training - A Technical Training Program To Be Qualified Pharmacovigilance Professional (QPPV)" training has been added to ResearchAndMarkets.com's offering.
  • QPPV stands for Qualified Person Responsible for Pharmacovigilance and is a Qualified and experienced professional in all aspects of Pharmacovigilance.
  • QPPV is required in Pharmacovigilance system as per the legal obligations in EU per GVP Module I which states that all marketing authorization holders to have one qualified person for pharmacovigilance (QPPV) with responsibility for establishing and maintaining all aspects of the company's global pharmacovigilance system.
  • The QPPV is responsible for all Pharmacovigilance related activities like risk management including detection, assessment, understanding and communication of safety information.

Recordati Rare Diseases now holds Isturisa® European MA

Wednesday, April 8, 2020 - 12:01pm

Paris, 8 April, 2020 Recordati Rare Diseases today announces the transfer of the European Marketing Authorization of Isturisa (osilodrostat).

Key Points: 
  • Paris, 8 April, 2020 Recordati Rare Diseases today announces the transfer of the European Marketing Authorization of Isturisa (osilodrostat).
  • The European Commission granted Isturisa a European Marketing Authorisation as an Orphan Drug.
  • Recordati Rare Diseases, part of the Recordati group, recently launched an endocrinology business unit and is actively building its commercial, medical, and market access teams.
  • Recordati is committed to the research and development of new specialties with a focus on treatments for rare diseases.

Alnylam Completes Rolling Submission of New Drug Application to the U.S. Food and Drug Administration and Submits Marketing Authorization Application to the European Medicines Agency for Lumasiran for the Treatment of Primary Hyperoxaluria Type 1

Tuesday, April 7, 2020 - 12:00pm

The Company also announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for lumasiran for the treatment of PH1.

Key Points: 
  • The Company also announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for lumasiran for the treatment of PH1.
  • Lumasiran has been granted Priority Medicines (PRIME) Designation by the EMA as well as Orphan Drug Designation in the European Union.
  • Accelerated assessment potentially provides a reduced review timeline from 210 to 150 days once the MAA is filed and validated.
  • Lumasiran is an investigational, subcutaneously administered RNAi therapeutic targeting glycolate oxidase (GO), in development for the treatment of primary hyperoxaluria type 1 (PH1).

Working Through Veterinary Drug Development in the EU and USA: 4-Day Course (London, United Kingdom - June 8-11, 2020) - ResearchAndMarkets.com

Monday, March 9, 2020 - 9:35am

The "Working Through Veterinary Drug Development in the EU and USA" conference has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Working Through Veterinary Drug Development in the EU and USA" conference has been added to ResearchAndMarkets.com's offering.
  • This intensive four-day course provides a thorough understanding of how to develop veterinary medicine in the EU and USA.
  • It will take participants through all the stages in the development of a veterinary medicinal product for which marketing authorisation is sought in the EU and the USA.
  • Presentations will cover pharmaceutical and process development, toxicological, pharmacological, residues and environmental studies, safety risk assessments, clinical development, regulatory and marketing input and project management.

3-Day Seminar: Pharmaceutical Regulatory Affairs in Asia (London, United Kingdom - April 15-17, 2020) - ResearchAndMarkets.com

Thursday, March 5, 2020 - 10:10am

The "Pharmaceutical Regulatory Affairs in Asia" conference has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Pharmaceutical Regulatory Affairs in Asia" conference has been added to ResearchAndMarkets.com's offering.
  • The pharmaceutical market in Asia is growing at a rapid pace and presents both opportunities and challenges to those wishing to work in the region.
  • Rather than a single market, Asia is a collection of different markets, each with their own regulatory processes, although harmonisation exists within the ASEAN countries.
  • This seminar will provide a practical overview of the key areas of pharmaceutical regulatory affairs in Asia, including India, and will cover all important aspects of gaining and maintaining a successful marketing authorisation within the region.

1-Day Clinical Overview & Summary Seminar: Creating an Effective Marketing Authorisation Application (London, UK - March 31st, 2020) - ResearchAndMarkets.com

Wednesday, March 4, 2020 - 2:56pm

The "Clinical Overview and Clinical Summary: Creating Effective Marketing Authorisation Application" conference has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Clinical Overview and Clinical Summary: Creating Effective Marketing Authorisation Application" conference has been added to ResearchAndMarkets.com's offering.
  • The Common Technical Document (CTD) Guideline is the obligatory format in the EU and most territories worldwide for registration applications.
  • The clinical overview and clinical summaries in Module 2 provide a critical analysis of the clinical data within the CTD.
  • This interactive course will present the regulatory guidelines and requirements of Module 2 and discuss practical approaches to developing the content and preparation of the clinical overview and clinical summaries.

Variations to Marketing Authorisations - 2 Day Course (London, United Kingdom - October 8-9, 2020) - ResearchAndMarkets.com

Tuesday, March 3, 2020 - 7:12pm

The "Variations to Marketing Authorisations" conference has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Variations to Marketing Authorisations" conference has been added to ResearchAndMarkets.com's offering.
  • It is a key post-marketing responsibility of the marketing authorisation holder (MAH) to keep dossiers up to date, and changes must be submitted as variations to the appropriate regulatory authority.
  • The programme will include filing tips and strategies with information and advice on creating a global dossier and implementation periods.
  • Practical sessions throughout the two days will provide an opportunity to discuss different scenarios and strategies with other participants.