Priority review

GenFleet Therapeutics Announces GFH009 Granted with FDA Fast Track, Orphan Drug Designations for Treating R/R Peripheral T-cell Lymphomas and Acute Myeloid Leukemia

Retrieved on: 
Thursday, January 11, 2024
Oncogene, FTD, MYC, Patient, Therapy, Accelerated approval (FDA), Acute myeloid leukemia, Venetoclax, Food and Drug Administration, ODD, Azacitidine, Food, MCL1, NDA, Safety, Priority review, FDA, BLA, Marketing, AML, Bone marrow, PTCL, CDK9, Pharmaceutical industry

GFH009 (highly selective CDK9 inhibitor) so far has received both fast track and orphan drug designations from the FDA for the treatment of adult patients with relapsed/refractory (r/r) peripheral T-cell lymphomas (PTCL) and r/r acute myeloid leukemia (AML).

Key Points: 
  • GFH009 (highly selective CDK9 inhibitor) so far has received both fast track and orphan drug designations from the FDA for the treatment of adult patients with relapsed/refractory (r/r) peripheral T-cell lymphomas (PTCL) and r/r acute myeloid leukemia (AML).
  • Numerous subjects in GFH009 studies achieved complete or partial responses, and superior safety profile & efficacy of GFH009 was observed in previous studies.
  • GenFleet is conducting a multi-center, open-label, single-arm study of GFH009 for the treatment of r/r PTCL in close to 40 sites in China.
  • GFH925 was granted with NDA acceptance, priority review and two breakthrough therapy designations in China; GFH009 received FDA fast track and orphan drug designations for two indications.

Abeona Therapeutics Announces $50 Million Credit Facility

Retrieved on: 
Monday, January 8, 2024
Marketing, Food, Concha bullosa, Priority review, FDA, PDUFA, PZ, RDEB, Biologics license application, Patient, COL7A1

CLEVELAND, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that it has entered into a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P.

Key Points: 
  • CLEVELAND, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that it has entered into a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P.
  • The credit agreement, which has a term of three and a half years, includes a first tranche of $20 million at closing, a second tranche of $10 million of committed capital, and an additional accordion option to upsize the credit facility by an additional $20 million upon satisfaction of certain terms and conditions.
  • The FDA has accepted and granted Priority Review with a PDUFA target action date of May 25, 2024 for the Biologics License Application for pz-cel.
  • “We are excited to enter into this relationship with Avenue Venture Fund and secure additional financial resources to further support our launch and commercialization efforts for pz-cel,” said Joe Vazzano, Chief Financial Officer of Abeona.

INOVIO Plans to Submit a BLA for INO-3107 as a Potential Treatment for RRP in Second Half of 2024 Under Accelerated Approval Program

Retrieved on: 
Wednesday, January 3, 2024
European Commission, Food, Priority review, Breakthrough therapy, FDA, Type, Health, DNA, Cancer, RRP, Recurrence, Biologics license application, Patient, Infection, Food and Drug Administration, BLA, Pharmaceutical industry

This announcement follows an Initial Comprehensive Multidisciplinary Breakthrough Therapy (Type B) Meeting with the FDA on critical aspects of the data package required to submit a BLA under the agency's accelerated approval program.

Key Points: 
  • This announcement follows an Initial Comprehensive Multidisciplinary Breakthrough Therapy (Type B) Meeting with the FDA on critical aspects of the data package required to submit a BLA under the agency's accelerated approval program.
  • "Based on productive discussions with the FDA, we believe we now have established a path to submitting a BLA for INO-3107 under the accelerated approval program," said Dr. Jacqueline Shea, INOVIO's President & Chief Executive Officer.
  • "Our plan is to complete the submission of our BLA in the second half of 2024 and request a Priority Review.
  • Concurrently, we will continue advancing our commercial plans, with the goal of being ready to launch INO-3107 in 2025."

MAPS PBC Announces New American Medical Association CPT III Codes for Psychedelic-Assisted Therapies Take Effect

Retrieved on: 
Tuesday, January 2, 2024
Continuity, Food, Priority review, Breakthrough therapy, Physician, MAPS, FDA, Intervention, Psychotherapy, NDA, SAN, MDMA, Mental health, PTSD, American Medical Association, Post-traumatic stress disorder, DEA, CPT, Safety, Pharmaceutical industry

SAN JOSE, Calif., Jan. 2, 2024 /PRNewswire/ -- MAPS Public Benefit Corporation ("MAPS PBC"), a clinical-stage company dedicated to changing the way mental health conditions are treated, announced that the American Medical Association ("AMA") current procedural terminology ("CPT") III codes for psychedelic-assisted therapies, first announced in July 2023, are now in effect. The new CPT codes will provide physicians and other qualified healthcare providers a means to seek coverage and reimbursement for delivering psychedelic-assisted therapy if approved by the U.S. Food and Drug Administration ("FDA").

Key Points: 
  • SAN JOSE, Calif., Jan. 2, 2024 /PRNewswire/ -- MAPS Public Benefit Corporation ("MAPS PBC"), a clinical-stage company dedicated to changing the way mental health conditions are treated, announced that the American Medical Association ("AMA") current procedural terminology ("CPT") III codes for psychedelic-assisted therapies, first announced in July 2023, are now in effect.
  • MAPS PBC and COMPASS Pathways collaborated to work with the AMA to create these new CPT III codes.
  • On March 3, 2023, the AMA published the Summary of Panel Actions which included the acceptance of the new CPT III codes.
  • With Breakthrough Therapy designation given to MDMA in 2017, MAPS PBC has requested the FDA grant Priority Review of the NDA.

U.S. Food and Drug Administration issues Complete Response Letter for dasiglucagon in congenital hyperinsulinism for up to three weeks of dosing due to inspection findings at third-party manufacturing facility

Retrieved on: 
Saturday, December 23, 2023
Food, Priority review, CGM, Symantec Endpoint Protection, FDA, NDA, New Drug Application, Congenital hyperinsulinism, Part, CRL, Patient, Safety, Hypoglycemia, Pharmaceutical industry

The CRL is related to deficiencies identified at a third-party manufacturing facility following an inspection but are not specific to dasiglucagon.

Key Points: 
  • The CRL is related to deficiencies identified at a third-party manufacturing facility following an inspection but are not specific to dasiglucagon.
  • The CRL is related to deficiencies identified following an inspection at a third-party contract manufacturing facility.
  • The FDA granted dasiglucagon Priority Review on August 30, 2023 for up to three weeks of dosing.
  • Part 1 relates to dosing of up to 3 weeks and Part 2 relates to the use beyond 3 weeks.

Humacyte Submits Biologics License Application (BLA) to U.S. FDA Seeking Approval of Human Acellular Vessel™ (HAV™) for the Treatment of Vascular Trauma

Retrieved on: 
Tuesday, December 12, 2023
Food, Priority review, Infection, HUMA, Injury, FDA, File, HAV, Tissue, MD, Doctor of Philosophy, Biologics license application, Defense, Amputation, Public law, Patient, Food and Drug Administration, BLA, Pharmaceutical industry

DURHAM, N.C., Dec. 12, 2023 (GLOBE NEWSWIRE) -- Humacyte, Inc. (Nasdaq: HUMA), a clinical-stage biotechnology platform company developing universally implantable, bioengineered human tissue at commercial scale, today announced that it has submitted a Biologics License Application (BLA) to U.S. Food and Drug Administration (FDA) seeking approval of the Human Acellular Vessel (HAV) in urgent arterial repair following extremity vascular trauma when synthetic graft is not indicated, and when autologous vein use is not feasible. The BLA submission is supported by positive results from the V005 Phase 2/3 clinical trial as well as from the treatment of wartime injuries in Ukraine. The HAV was observed to have higher rates of patency (blood flow), and lower rates of amputation and infection, as compared to historic synthetic graft benchmarks.

Key Points: 
  • The BLA submission is supported by positive results from the V005 Phase 2/3 clinical trial as well as from the treatment of wartime injuries in Ukraine.
  • The HAV was observed to have higher rates of patency (blood flow), and lower rates of amputation and infection, as compared to historic synthetic graft benchmarks.
  • Humacyte has requested Priority Review of the application and, if granted, the review should be completed within six months of the filing acceptance date.
  • In May 2023 the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for use of the HAV in urgent arterial repair following extremity vascular trauma.

Orchard Therapeutics Receives Swissmedic Approval for Libmeldy in Early-onset MLD

Retrieved on: 
Monday, December 11, 2023
Nerve, MLD, Traffic barrier, European Commission, Food, Agency, Priority review, Health care, Death, FDA, PDUFA, Civil service commission, HSC, MHRA, Therapy, Busulfan, ARSA, Mutation, Atidarsagene autotemcel, Genome, Central nervous system, Patient, Health, Pharmaceutical industry

BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD).

Key Points: 
  • BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD).
  • Libmeldy aims to correct the underlying genetic cause of MLD by inserting a working copy of the ARSA gene into the genome of a patients’ own HSCs.
  • With more than a cumulative 250 patient-years of follow-up, Libmeldy was generally well-tolerated, with no treatment-related serious adverse events or deaths.
  • “Today’s approval of Libmeldy in Switzerland opens up tremendous new possibilities for eligible children with MLD who previously had no approved treatment options beyond supportive care,” said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics.

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.

Genelux Corporation Receives FDA Fast Track Designation for Olvi-Vec in Platinum Resistant/Refractory Ovarian Cancer

Retrieved on: 
Monday, November 27, 2023
Platinum, Priority review, Ovarian cancer, FDA, Genelux Corporation, Patient, Safety, Fast Track, Pharmaceutical industry

WESTLAKE VILLAGE, Calif., Nov. 27, 2023 (GLOBE NEWSWIRE) -- Genelux Corporation (NASDAQ: GNLX), a late clinical-stage immuno-oncology company, today announced that the United States Food and Drug Administration (FDA) has granted Fast Track designation for the development program of Olvi-Vec (olvimulogene nanivacirepvec) for the treatment of patients with platinum resistant/refractory ovarian cancer.

Key Points: 
  • WESTLAKE VILLAGE, Calif., Nov. 27, 2023 (GLOBE NEWSWIRE) -- Genelux Corporation (NASDAQ: GNLX), a late clinical-stage immuno-oncology company, today announced that the United States Food and Drug Administration (FDA) has granted Fast Track designation for the development program of Olvi-Vec (olvimulogene nanivacirepvec) for the treatment of patients with platinum resistant/refractory ovarian cancer.
  • “The Fast Track designation granted for Olvi-Vec underscores its potential to address unmet medical needs in ovarian cancer, a significant recognition as we continue to enroll our Phase 3 OnPrime study,” said Thomas Zindrick , President, Chairman and CEO of Genelux.
  • The purpose of Fast Track designation is to facilitate the development and hasten the review process of drugs aimed at treating serious and life-threatening conditions, ensuring that an approved product can swiftly enter the market.
  • Notable aspects of Fast Track designation encompass regular engagements with the FDA review team, and, if specific criteria are satisfied, potential eligibility for Priority Review and Rolling Review.

Abeona Therapeutics Announces FDA Accepts and Grants Priority Review for Pz-cel Biologics License Application (BLA)

Retrieved on: 
Monday, November 27, 2023
Quality of life, Priority review, Food, Concha bullosa, ISID, BLA, FDA, PDUFA, Biologics license application, Advisory Committee, Patient, Safety, Wound healing, COL7A1, Pharmaceutical industry

CLEVELAND, Nov. 27, 2023 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review for the Biologics License Application (BLA) for pz-cel (prademagene zamikeracel), Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of May 25, 2024. The FDA also advised that it does not currently plan to convene an Advisory Committee meeting to discuss the pz-cel application.

Key Points: 
  • Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of May 25, 2024.
  • The FDA also advised that it does not currently plan to convene an Advisory Committee meeting to discuss the pz-cel application.
  • Both studies revealed that a one-time application of pz-cel on large and chronic wounds delivered sustained wound healing and pain reduction.
  • The grant of the Priority Review status is an important prerequisite for Abeona’s eligibility for a Priority Review Voucher.