Marketing authorization

Orchard Therapeutics Reports Third Quarter 2019 Financial Results and Achievement of Key 2019 Milestones for Neurometabolic and Primary Immune Deficiencies Franchises

Wednesday, November 6, 2019 - 9:01pm

We are working with care and urgency to make these therapies broadly available to patients who need them.

Key Points: 
  • We are working with care and urgency to make these therapies broadly available to patients who need them.
  • The company is on-track for a near-term Marketing Authorization Application (MAA) submission for OTL-200.
  • -- The study is expected to enroll eight patients by the first half of 2020, with primary endpoint results expected after one year of follow-up.
  • The company had 97.8 million weighted average ordinary shares outstanding for the three months ended September 30, 2019.

Aimmune Therapeutics Announces Third Quarter 2019 Financial Results and Provides Operational Highlights

Wednesday, November 6, 2019 - 9:05pm

Cash, cash equivalents, and investments totaled $200.5 million on September 30, 2019, compared to $303.9 million on December 31, 2018.

Key Points: 
  • Cash, cash equivalents, and investments totaled $200.5 million on September 30, 2019, compared to $303.9 million on December 31, 2018.
  • In connection with this announcement, Aimmune Therapeutics will host a conference call and webcast today at 4:30 p.m.
  • To access a live or recorded webcast of the call, please visit the Investor Relations section of the Aimmune Therapeutics website at www.aimmune.com .
  • The European Medicines Agency (EMA) is reviewing Aimmunes Marketing Authorization Application (MAA) for PALFORZIA, which Aimmune submitted in June 2019.

MediWound to Report Third Quarter 2019 Financial Results and Host a Conference Call and Webcast on November 14

Wednesday, November 6, 2019 - 1:00pm

To access the call, participants should dial the applicable telephone number above at least 5 minutes prior to the start of the call.

Key Points: 
  • To access the call, participants should dial the applicable telephone number above at least 5 minutes prior to the start of the call.
  • An archived version of the webcast will be available for replay for 90 days in the Investors section of the MediWound website.
  • The product has received marketing authorization from the European Medicines Agency as well as the Israeli, Argentinian, South Korean, Russian and Peruvian Ministries of Health.
  • In two Phase 2 studies, EscharEx has demonstrated safety and efficacy in the debridement of various chronic and other hard-to-heal wounds, within a few daily applications.

Vertex Announces European Medicines Agency Marketing Authorization Application Validation for VX-445 (Elexacaftor), Tezacaftor and Ivacaftor Triple Combination Treatment in Cystic Fibrosis

Thursday, October 31, 2019 - 11:21am

Vertex Pharmaceuticals (Europe) Limited today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen.

Key Points: 
  • Vertex Pharmaceuticals (Europe) Limited today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen.
  • In these studies, the triple combination regimen was generally well tolerated.
  • Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.
  • Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

Aimmune Therapeutics to Host Conference Call and Webcast to Discuss Third Quarter 2019 Financial Results and Recent Operational Highlights

Wednesday, October 30, 2019 - 11:00am

ET to discuss its financial results for the quarter ended September 30, 2019, and recent operational highlights.

Key Points: 
  • ET to discuss its financial results for the quarter ended September 30, 2019, and recent operational highlights.
  • To access the live call by phone, dial (877) 497-1438 (domestic) or (262) 558-6296 (international) and enter the passcode 7842939.
  • To access a live or recorded webcast of the call, please visit the Investor Relations section of the Aimmune Therapeutics website at www.aimmune.com .
  • The European Medicines Agency (EMA) is reviewing Aimmunes Marketing Authorization Application (MAA) for PALFORZIA, which Aimmune submitted in June 2019.

Basilea reports marketing authorization of antifungal Cresemba® in Brazil

Tuesday, October 29, 2019 - 6:15am

(GBT), received the regulatory approval and marketing authorization for the antifungal Cresemba (isavuconazole) in Brazil.

Key Points: 
  • (GBT), received the regulatory approval and marketing authorization for the antifungal Cresemba (isavuconazole) in Brazil.
  • David Veitch, Basilea's Chief Executive Officer, said: We are very pleased with the approval of Cresemba in Brazil, which is commercially the most important market for the brand in Latin America.
  • We are looking forward to GBT launching Cresemba in Brazil, adding yet another market towards our goal of doubling the number of countries in which Cresemba is launched to 40 by the end of 2019.
  • Cresemba was reviewed under the recently introduced orphan drug legislation in Brazil and has U.S. and European orphan drug designation for its approved indications.

European Medicines Agency Approves Refined Commercial Manufacturing Specifications for ZYNTEGLO™

Tuesday, October 22, 2019 - 12:30pm

The refined commercial drug product specifications support the efficacy and safety profile of ZYNTEGLO and will give patients the best opportunity for clinically meaningful outcomes consistent with the results that were foundational to the conditional marketing authorization in the European Union.

Key Points: 
  • The refined commercial drug product specifications support the efficacy and safety profile of ZYNTEGLO and will give patients the best opportunity for clinically meaningful outcomes consistent with the results that were foundational to the conditional marketing authorization in the European Union.
  • ZYNTEGLO addresses the underlying genetic cause of TDT and offers patients the potential to become transfusion independent, which once achieved is expected to be life-long.
  • We are proud to be the commercial manufacturing partner of bluebird bio and to be part of bringing this potentially life-changing therapy to TDT patients in Europe.
  • ZYNTEGLO has received a conditional marketing authorization in the European Union and is not approved in the United States.

FDA Approves TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older Who Have at Least One F508del Mutation

Wednesday, October 23, 2019 - 3:08pm

We remain committed to relentlessly pursuing the development of transformative therapies for all people living with this disease.

Key Points: 
  • We remain committed to relentlessly pursuing the development of transformative therapies for all people living with this disease.
  • Vertex has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the elexacaftor/tezacaftor/ivacaftor combination regimen.
  • It is not known if TRIKAFTA is safe and effective in children under 12 years of age.
  • TRIKAFTA can cause serious side effects, including:
    High liver enzymes in the blood, which is a common side effect in people treated with TRIKAFTA.

QPPV Online Training 2019: A Technical Training Program to be a Qualified Pharmacovigilance Professional (QPPV) - ResearchAndMarkets.com

Thursday, October 17, 2019 - 5:01pm

This will be a perfect learning opportunity in all areas of drug safety, pharmacovigilance, regulatory and quality compliance and will be a great addition to your knowledge in pharmacovigilance.

Key Points: 
  • This will be a perfect learning opportunity in all areas of drug safety, pharmacovigilance, regulatory and quality compliance and will be a great addition to your knowledge in pharmacovigilance.
  • 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.

First Algenex CrisBio®-based vaccine submitted to European Medicines Agency

Tuesday, October 15, 2019 - 7:00am

Madrid (Spain), October 15, 2019 Algenex SL, a private biotechnology company delivering disruptive technologies for recombinant biologics production, today announces that one of its industry partners has submitted a dossier to the European Medicines Agency (EMA) for a recombinant VLP vaccine produced using Algenex proprietary CrisBio technology.

Key Points: 
  • Madrid (Spain), October 15, 2019 Algenex SL, a private biotechnology company delivering disruptive technologies for recombinant biologics production, today announces that one of its industry partners has submitted a dossier to the European Medicines Agency (EMA) for a recombinant VLP vaccine produced using Algenex proprietary CrisBio technology.
  • The submission is targeting a European marketing authorization via the centralized approval system.
  • This is the first vaccine produced using Algenex CrisBio technology to be submitted for regulatory approval.
  • CrisBio is Algenex proprietary and patent protected Baculovirus vector-mediated expression platform that harnesses the power of insects to act as natural single-use bioreactors.