MAA

Outlook Therapeutics® Reports Financial Results for Fiscal Year 2023 and Reiterates ONS-5010 Clinical and Regulatory Path Forward in the U.S. and EU

Retrieved on: 
Friday, December 22, 2023
SPA, Ranibizumab, Partnership, Food, Outlook, FDA, MAA, EMA, Completion, Retina, AMD, Injection, Trial of the century, Therapy, Bevacizumab, Calendar, Protocol, European Medicines Agency, Feedback, AmerisourceBergen, Committee, Patient, Food and Drug Administration, CHMP, Pharmaceutical industry

Subsequently, as discussed with and recommended by the FDA, Outlook Therapeutics submitted a clinical trial protocol and requested a Type A meeting with the FDA for feedback.

Key Points: 
  • Subsequently, as discussed with and recommended by the FDA, Outlook Therapeutics submitted a clinical trial protocol and requested a Type A meeting with the FDA for feedback.
  • The FDA has already provided written feedback on the protocol, which Outlook Therapeutics has incorporated.
  • As of September 30, 2023, Outlook Therapeutics had cash and cash equivalents of $23.4 million.
  • On December 21, 2023, Outlook Therapeutics reached an agreement with the holder of its outstanding convertible promissory note to extend the maturity until April 1, 2024.

Apellis Provides Update on Ongoing Regulatory Review of Pegcetacoplan for GA in the European Union

Retrieved on: 
Thursday, December 14, 2023
Quality of life, Geographic atrophy, EMA, MAA, AMD, OAKS, European Medicines Agency, Webcast, Committee, Patient, Paratriathlon, Retina, CHMP, APLS

Following the oral explanation meeting on December 13, Apellis was informed of a negative trend vote on the MAA for pegcetacoplan.

Key Points: 
  • Following the oral explanation meeting on December 13, Apellis was informed of a negative trend vote on the MAA for pegcetacoplan.
  • Apellis expects the CHMP to adopt a negative opinion at its next meeting, which is scheduled to be held January 22-25, 2024.
  • If a negative opinion is adopted, Apellis plans to appeal the outcome and seek re-examination of the opinion.
  • Treatment with both every-other-month and monthly pegcetacoplan reduced GA lesion growth with increasing treatment effects over time and showed a well-demonstrated safety profile.

TFF Pharmaceuticals Appoints Thomas B. King to Board of Directors

Retrieved on: 
Wednesday, December 13, 2023
McPherson College, Business, Marketing, Chemistry, MAA, NDA, MBA, Bachelor of Arts, Cephalon, University, Abbott Laboratories, Laboratory, IPO, Patient, TFF, Management

King to its Board of Directors.

Key Points: 
  • King to its Board of Directors.
  • “I am pleased to welcome Thomas King to our Board of Directors,” said Harlan Weisman, M.D., Vice Chairman and Chief Executive Officer of TFF Pharmaceuticals.
  • “Tom is an exceptionally accomplished executive who has held leadership positions at life sciences companies over the last three decades.
  • Presently, Mr. King sits on the board of several companies, including Achieve Life Sciences (Nasdaq: ACHV), Kinaset Therapeutics, and Concentric Analgesics.

GENFIT Updates 2024 Outlook Following Acceptance of Elafibranor Filings in Primary Biliary Cholangitis (PBC)

Retrieved on: 
Friday, December 8, 2023
Hepatic encephalopathy, Ipsen, AASLD, Cell death, Food, Itch, Priority review, Food and Drug Administration, PSC, IND, ACLF, Marketing, FDA, EMA, MAA, PDUFA, Permeability (electromagnetism), Liver, NDA, Inflammation, Primary sclerosing cholangitis, ALP, Capital market, Hyperammonemia, Patient, NTZ, Fibrosis, PBC, ASK1

Launches in the US and Europe will also make GENFIT eligible for royalty payments.

Key Points: 
  • Launches in the US and Europe will also make GENFIT eligible for royalty payments.
  • Revenues will fund the development of GENFIT’s pipeline, now mainly focused on Acute On-Chronic Liver Failure (ACLF) with 5 differentiated assets.
  • Acceptance of filings in the US and Europe have triggered the first milestone payment for GENFIT.
  • Ipsen also indicated that it is developing elafibranor in Primary Sclerosing Cholangitis (PSC) which could lead to further revenues for GENFIT under the licensing agreement.

Ipsen confirms U.S. FDA grants priority review for New Drug Application for elafibranor for the treatment of rare cholestatic liver disease, PBC

Retrieved on: 
Thursday, December 7, 2023
Liver, Ascending cholangitis, Ipsen, Food, Itch, Health care, Woman, FDA, EMA, MAA, PDUFA, Primary biliary cholangitis, Bile, Fatigue, NDA, Elafibranor, MHRA, ADR, New Drug Application, European Medicines Agency, Common, IPN, PPAR, Liver failure, Patient, Medicine, EVP, PBC, CHMP, Pharmaceutical industry

New Drug Application granted priority review with PDUFA date set for June 10, 2024

Key Points: 
  • New Drug Application granted priority review with PDUFA date set for June 10, 2024
    Investigational elafibranor is the first novel second-line treatment for primary biliary cholangitis (PBC) to be filed in E.U.
  • An oral, once-daily dual peroxisome activated receptor alpha/delta (PPAR α,δ) agonist, investigational elafibranor could potentially be the first novel second-line treatment for the rare, cholestatic liver disease, PBC, in nearly a decade.
  • The target FDA PDUFA date under priority review is June 10, 2024.
  • “This is a condition where many patients are living with worsening disease and debilitating symptoms despite being on treatment.

Ipsen confirms U.S. FDA grants priority review for New Drug Application for elafibranor for the treatment of rare cholestatic liver disease, PBC

Retrieved on: 
Thursday, December 7, 2023
Liver, UDCA, Chapter Two, Woman, FDA, MAA, Fatigue, Nausea, Primary biliary cholangitis, Inflammation, ADR, Patent, Liver disease, Diagnosis, AMF, CHMP, Ascending cholangitis, UCD, Business, ACLF, PDUFA, Itch, Risk, New Drug Application, OA, Common, EASL, MASH, Liver failure, Safety, Ammonia, Elafibranor, Therapy, Epidemiology, History, Patient, Cholangiocarcinoma, Food, Health care, EMA, Ursodeoxycholic acid, MHRA, Cirrhosis, Medicine, NTZ, EVP, Cholestasis, NASH, Research, Diarrhea, Bile, NDA, PBC, Abdominal pain, Clinical trial, European Medicines Agency, IPN, PPAR, P10, CCA, Fibrosis, Pharmaceutical industry

An oral, once-daily dual peroxisome activated receptor alpha/delta (PPAR α,δ) agonist, investigational elafibranor could potentially be the first novel second-line treatment for the rare, cholestatic liver disease, PBC, in nearly a decade.

Key Points: 
  • An oral, once-daily dual peroxisome activated receptor alpha/delta (PPAR α,δ) agonist, investigational elafibranor could potentially be the first novel second-line treatment for the rare, cholestatic liver disease, PBC, in nearly a decade.
  • The trial enrolled 161 patients who were randomized 2:1 to receive either elafibranor 80mg once daily or placebo.
  • ELATIVE also investigated the effect of treatment with elafibranor on pruritus (severe itch), a significant symptom burden amongst people living with PBC.
  • Findings from the secondary endpoint using the PBC Worst Itch NRS score, showed a reduction in pruritis for elafibranor, which was not statistically significant.

Outlook Therapeutics® Announces Strategic Organizational Realignment

Retrieved on: 
Wednesday, December 6, 2023
Senior, SPA, Ranibizumab, Partnership, Food, Senior advisor, Symantec Endpoint Protection, Marketing, FDA, MAA, EMA, Acquisition, Type, Retina, CMC, Therapy, Bevacizumab, Calendar, European Medicines Agency, CRL, Committee, Patient, Food and Drug Administration, CHMP, BLA

Effective immediately, Joel Prieve, formerly the Senior Vice President of Commercial Operations, has been appointed to the role of Senior Vice President of Licensing and M&A.

Key Points: 
  • Effective immediately, Joel Prieve, formerly the Senior Vice President of Commercial Operations, has been appointed to the role of Senior Vice President of Licensing and M&A.
  • Previously, Outlook Therapeutics announced the addition of Jedd Comiskey as Senior Vice President – Head of Europe to focus on potential commercial launch and commercial partnership opportunities for ONS-5010 in the EU, if approved.
  • As previously reported, Outlook Therapeutics is working with the FDA to design an additional adequate and well-controlled clinical trial to support the planned ONS-5010 BLA resubmission.
  • Outlook Therapeutics also submitted a Marketing Authorization Application (MAA) in Europe, which was validated for review in December 2022.

Pfizer and Valneva Complete Recruitment for Phase 3 VALOR Trial for Lyme Disease Vaccine Candidate, VLA15

Retrieved on: 
Monday, December 4, 2023
DSMB, Food, Șaeș, Euronext Paris, BLA, Data monitoring committee, Marketing, PFE, FDA, EMA, MAA, Lyme disease, Disease, Research, Biologics license application, European Medicines Agency, VLA, Safety, Development, Senior, Vaccine

Pfizer and Valneva Complete Recruitment for Phase 3 VALOR Trial for Lyme Disease Vaccine Candidate, VLA15

Key Points: 
  • Pfizer and Valneva Complete Recruitment for Phase 3 VALOR Trial for Lyme Disease Vaccine Candidate, VLA15
    9,437* participants enrolled at sites across the U.S., Europe and Canada in areas where Lyme disease is endemic
    Pfizer aims to submit regulatory filings in the U.S. and Europe in 2026
    New York, NY, and Saint-Herblain (France), December 4, 2023 – Pfizer Inc. (NYSE: PFE) and Valneva SE (Nasdaq: VALN; Euronext Paris: VLA) today announced that they have completed recruitment for the Phase 3 trial Vaccine Against Lyme for Outdoor Recreationists (VALOR) (NCT05477524) for Lyme disease vaccine candidate VLA15.
  • The trial builds on previous positive Phase 1 and 2 trial results and includes both adult and pediatric participants, with the aim to confirm the efficacy, safety, lot consistency, and immunogenicity of VLA15.
  • “We are pleased that the Phase 3 trial recruitment is complete.
  • Pfizer and Valneva entered into a collaboration agreement in April 2020 to co-develop VLA15, with updates to the terms within this agreement made in June 2022.3,4

Krystal Biotech Announces EMA Validation of Marketing Authorization Application for VYJUVEK for the Treatment of Dystrophic Epidermolysis Bullosa

Retrieved on: 
Monday, November 27, 2023
Research and development, Marketing, Suma, Concha bullosa, EMA, MAA, Orphan, DEB, KRYS, Committee, Patient, CHMP

PITTSBURGH, Nov. 27, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, today announced that the Company’s Marketing Authorization Application (MAA) to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for VYJUVEK (beremagene geperpavec-svdt, also known as B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB) has been validated and is now under CHMP review.

Key Points: 
  • PITTSBURGH, Nov. 27, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, today announced that the Company’s Marketing Authorization Application (MAA) to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for VYJUVEK (beremagene geperpavec-svdt, also known as B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB) has been validated and is now under CHMP review.
  • A CHMP opinion is anticipated in the second half of 2024.
  • Based on this positive opinion, the Company would be eligible for up to an additional two years of marketing exclusivity in the EU, on top of the ten-year EU market exclusivity after market approval in the EU.
  • Previously, VYJUVEK received Orphan Drug Designation and PRIority Medicines (PRIME) eligibility from the EMA.

EMA Accepts Valneva’s Chikungunya Vaccine Marketing Authorization Application for Accelerated Assessment

Retrieved on: 
Monday, November 27, 2023
Licensure, Food, Risk, Euronext Paris, Saint-Herblain, Marketing, EMA, MAA, FDA, Disease, MD, Chikungunya, European Medicines Agency, Public, VLA, CHMP, Pharmaceutical industry

Saint-Herblain (France), November 27, 2023 – Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, today announced that the European Medicines Agency (EMA) has performed a technical validation of the Marketing Authorization Application (MAA) for Valneva’s single-shot chikungunya vaccine candidate VLA1553 and has determined that all essential regulatory elements required for scientific assessment were included in the application.

Key Points: 
  • Saint-Herblain (France), November 27, 2023 – Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, today announced that the European Medicines Agency (EMA) has performed a technical validation of the Marketing Authorization Application (MAA) for Valneva’s single-shot chikungunya vaccine candidate VLA1553 and has determined that all essential regulatory elements required for scientific assessment were included in the application.
  • The MAA was granted accelerated assessment1 last month by EMA’s Committee for Medicinal Products for Human Use (CHMP) based on the vaccine candidate’s “major interest for public health and therapeutic innovation”2.
  • Accelerated assessment reduces the timeframe for EMA’s CHMP to review a MAA once it is accepted for review from 210 days under the standard review procedure to 150 days.
  • The trial is also expected to support licensure of the vaccine in Brazil, which would be the first potential approval for use in endemic populations.