Priority review

Orchard Therapeutics Receives FDA Approval of Lenmeldy™ (atidarsagene autotemcel), the Only Therapy for Eligible Children with Early-onset Metachromatic Leukodystrophy in the U.S.

Retrieved on: 
Monday, March 18, 2024
Gastroenteritis, Genomics, Metabolism, Neutropenia, Brain, Nerve, Mutation, RPD, Disease, Incidence, Partnership, Central nervous system, Genetics, GSK, Stomatitis, Patient, Research, Fats, PRV, Traffic barrier, Child, Viral, Gene therapy, SAN, Genome, Urinary tract infection, Rash, Hepatomegaly, Priority review, Stem cell, Infection, Therapy, MLD, Kyowa Kirin, Febrile neutropenia, Fever, PSLI, Prognosis, Health, Family, FDA, San Raffaele Hospital, ARSA, Orchard, Food, Pharmaceutical industry

“MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.

Key Points: 
  • “MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.
  • This achievement is the culmination of decades of research and development in partnership with our academic and clinical collaborators at the San Raffaele-Telethon Institute for Gene Therapy.
  • It was previously given both Rare Pediatric Disease (RPD) and Regenerative Medicine Advanced Therapy (RMAT) designations from FDA.
  • Orchard Therapeutics will provide more details about the launch of Lenmeldy in the U.S. through a separate announcement this week.

Abeona Therapeutics Reports Full Year 2023 Financial Results and Announces Completion of FDA Inspections

Retrieved on: 
Monday, March 18, 2024
Research, AAV, Advisory Committee, Form FDA 483, PLI, Sale, EIR, University, Trial of the century, Rett syndrome, Concha bullosa, Priority review, Investment, IP, Therapy, Safety, REMS, EB, Food, FDA, BLA, RDEB, PDUFA, Pharmaceutical industry

On March 15, 2024, the Company submitted a response to the FDA, outlining already implemented and ongoing steps toward resolution that follow FDA guidance provided during the audit.

Key Points: 
  • On March 15, 2024, the Company submitted a response to the FDA, outlining already implemented and ongoing steps toward resolution that follow FDA guidance provided during the audit.
  • In January 2024, the FDA completed both a Bioresearch Monitoring (BIMO) inspection of Abeona and the Mid-Cycle Meeting regarding the pz-cel BLA.
  • Following completion of the BIMO inspection, the FDA inspector did not issue any observations or FDA Form 483s.
  • In March 2024, Abeona received a written Establishment Inspection Report (EIR), the formal report from the FDA regarding the BIMO inspection.

ENHERTU® Approved in the U.S. as First Tumor Agnostic HER2 Directed Therapy for Previously Treated Patients with Metastatic HER2 Positive Solid Tumors

Retrieved on: 
Saturday, April 6, 2024
Research, Women, FDA, Clinical Trials, Biotechnology, Pharmaceutical, Consumer, Health, Science, Oncology, Orbis, Urinary tract infection, Cardiac arrest, Stomatitis, Patient, Stroke, Lung cancer, University, Acute kidney injury, Neoplasm, Therapy, Anemia, Vomiting, Pleural effusion, Cellulitis, Shock (mechanics), Periodic breathing, Colorectal cancer, Abdominal pain, Safety, Immunohistochemistry, Fatigue, ADC, Daiichi Sankyo, Food, IHC, Sepsis, NSCLC, Appetite, Biliary tract, Constipation, NCCN, MD, Priority review, Fatality, Pneumonitis, HER2, ILD, Medication, Pneumonia, AstraZeneca, COVID-19, Biomarker, Diarrhea, Bleeding, MD Anderson Cancer Center, Nausea, Upper respiratory tract infection, Pharmaceutical industry

The first tumor agnostic approval of a HER2 directed therapy and ADC was based on efficacy data in 192 adult patients with previously treated unresectable or metastatic HER2 positive (IHC 3+) solid tumors who were enrolled in one of three multicenter phase 2 trials from the DESTINY clinical development program, including DESTINY-PanTumor02 , DESTINY-Lung01 or DESTINY-CRC02 .

Key Points: 
  • The first tumor agnostic approval of a HER2 directed therapy and ADC was based on efficacy data in 192 adult patients with previously treated unresectable or metastatic HER2 positive (IHC 3+) solid tumors who were enrolled in one of three multicenter phase 2 trials from the DESTINY clinical development program, including DESTINY-PanTumor02 , DESTINY-Lung01 or DESTINY-CRC02 .
  • In DESTINY-PanTumor02, efficacy was assessed in a subgroup of previously treated patients (n=111) with centrally or locally assessed HER2 positive (IHC 3+) solid tumors including either biliary tract, bladder, cervical, endometrial, ovarian, pancreatic or other tumors.
  • In DESTINY-Lung01, efficacy was assessed in a subgroup of patients (n=17) with centrally confirmed HER2 positive (IHC 3+) non-small cell lung cancer (NSCLC).
  • In DESTINY-CRC02, efficacy was assessed in the subgroup of patients (n=64) with centrally confirmed HER2 positive (IHC 3+) colorectal cancer.

Aditxt Signs a Definitive Agreement to Acquire Appili Therapeutics Inc., Developer of a Biodefense Vaccine Funded by the U.S. Department of Defense (DoD)

Retrieved on: 
Tuesday, April 2, 2024
Health, Defense, Infectious Diseases, Pharmaceutical, Contracts, Biotechnology, Ulcer, Paromomycin, Patient, NTD, IND, Metronidazole, Francisella tularensis, Infection, OTCQX, Therapy, Priority review, USD, Risk, Systemic, NIH, Anthrax, Arrangement, World Health Organization, Investigational New Drug, Calendar, Scar, Neglected tropical diseases, Tablet, Public, Research, News, PRV, Acquisition, TSX, Drug development, Population health, Cutaneous leishmaniasis, Canada Business Corporations Act, FDA, Transaction, Government, Growth, Entrepreneurship, Skin infection, Share, Procurement, WHO, Pharmaceutical industry

The U.S. FDA's approval of LIKMEZ highlighted Appili’s capacity to identify and develop significant opportunities within the infectious disease domain.

Key Points: 
  • The U.S. FDA's approval of LIKMEZ highlighted Appili’s capacity to identify and develop significant opportunities within the infectious disease domain.
  • Appili licensed the manufacturing and commercialization rights in the U.S. and other selected territories to Saptalis Pharmaceuticals, LLC (“Saptalis”).
  • Appili, in collaboration with Saptalis, continued the product's development, ultimately achieving FDA approval in the United States.
  • With FDA approval, future revenue is expected to be derived from milestone payments and royalties from Saptalis under the license agreement.

Nuvation Bio to Acquire AnHeart Therapeutics in All-Stock Transaction

Retrieved on: 
Monday, March 25, 2024
Oncology, Health, Clinical Trials, General Health, Pharmaceutical, Biotechnology, NUVB, Morrison & Foerster, Partner, NMPA, Cayman Islands, Entrepreneurship, NSCLC, Patient, Conyers, Georgia, LLP, Haiwen & Partners, Acquisition, Cancer, Cooley LLP, Priority review, Fangda, Intellectual property, FDA, ROS1, Cayman, Food, Pharmaceutical industry

Nuvation Bio Inc. (NYSE: NUVB), a biopharmaceutical company tackling some of the greatest unmet needs in oncology by developing differentiated and novel therapeutic candidates, and AnHeart Therapeutics Ltd. (AnHeart), a global clinical-stage biopharmaceutical company developing novel precision therapies for people with cancer, today announced that the companies have entered into a definitive agreement for Nuvation Bio to acquire AnHeart in an all-stock transaction (the Acquisition).

Key Points: 
  • Nuvation Bio Inc. (NYSE: NUVB), a biopharmaceutical company tackling some of the greatest unmet needs in oncology by developing differentiated and novel therapeutic candidates, and AnHeart Therapeutics Ltd. (AnHeart), a global clinical-stage biopharmaceutical company developing novel precision therapies for people with cancer, today announced that the companies have entered into a definitive agreement for Nuvation Bio to acquire AnHeart in an all-stock transaction (the Acquisition).
  • Immediately following the closing of the Acquisition, the former shareholders of AnHeart will own approximately 33% and the current stockholders of Nuvation Bio will own approximately 67% of Nuvation Bio on a fully diluted basis.
  • Subject to approval by the Nuvation Bio stockholders (the Nuvation Bio Stockholder Approval), each share of Series A Non-Voting Convertible Preferred Stock issued by Nuvation Bio in the Acquisition will initially be convertible into 100 shares of Class A common stock.
  • Any shareholders of AnHeart who are not accredited investors will receive cash for their AnHeart shares in lieu of receiving Nuvation Bio securities.

Takeda Announces U.S. FDA Approval of Supplemental New Drug Application (sNDA) for ICLUSIG® (ponatinib) in Adult Patients with Newly Diagnosed Ph+ ALL

Retrieved on: 
Tuesday, March 19, 2024
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, AP, Mutation, BP, Disease, New Drug Application, Patient, ALL, Shanda, CML, MD, University, MD Anderson Cancer Center, Priority review, MRD, CP, TKI, Glomus tumor, Food, Pharmaceutical industry

Takeda ( TSE:4502/NYSE:TAK ) today announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental New Drug Application (sNDA) for ICLUSIG® (ponatinib) for the treatment of adult patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in combination with chemotherapy.

Key Points: 
  • Takeda ( TSE:4502/NYSE:TAK ) today announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental New Drug Application (sNDA) for ICLUSIG® (ponatinib) for the treatment of adult patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in combination with chemotherapy.
  • This indication is approved under accelerated approval based on minimal residual disease (MRD)-negative complete remission (CR) at the end of induction.
  • Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
  • ICLUSIG is not indicated and is not recommended for the treatment of patients with newly diagnosed CP-CML.

Cybin Announces Positive End-of-Phase 2 Meeting with FDA for CYB003 in Major Depressive Disorder and Phase 3 Program Design

Retrieved on: 
Thursday, March 14, 2024
Mental Health, Finance, FDA, Clinical Trials, Professional Services, Biotechnology, Alternative Medicine, Health, Pharmaceutical, Depression, Week, DEA, BTD, Inc., MDD, Patient, Accelerated approval (FDA), DSM-IV codes, Suicidal ideation, Behavior, Priority review, Drug development, Major depressive disorder, Pharmaceutical industry

The Company has received minutes from its End-of-Phase-2 meeting with the FDA and reached alignment on its Phase 3 program design.

Key Points: 
  • The Company has received minutes from its End-of-Phase-2 meeting with the FDA and reached alignment on its Phase 3 program design.
  • The Company intends to commence enrollment for the multinational, multisite Phase 3 program in mid-year 2024.
  • The Company has engaged Worldwide Clinical Trials (“Worldwide”), a global, full-service contract research organization with deep expertise managing clinical trials for mental health conditions, including major depressive disorder.
  • Having aligned on key features of the pivotal program, we look forward to initiating a multisite, multinational Phase 3 program around mid-year,” said Doug Drysdale, Chief Executive Officer of Cybin.

FDA Approves New Antibiotic for Three Different Uses

Retrieved on: 
Wednesday, April 3, 2024
Daptomycin, Research, Hypersensitivity, Hypokalemia, Fungal infection, Aztreonam, Dizziness, Pneumonia, Survival, Dysgeusia, Potassium, Seizure, Abdominal pain, Dietary supplement, CABP, Headache, Patient, Public, Insomnia, Bacterial pneumonia, Food and Drug Administration, Animal, Bilirubin, Diarrhea, History, Mortality, Vancomycin, Leukopenia, Clostridioides, Human services, Injection site reaction, Nausea, Rash, Priority review, Bronchopneumonia, Skin, Fever, Heated tobacco product, Ceftriaxone, Safety, SAB, Anemia, Division, Phlebitis, Cosmetics, Vaccine, Infection, Food, Vomiting, Blood, Multimedia, FDA, Periodic breathing, ABSSSI, Pharmaceutical industry

"The FDA will continue our important work in this area as part of our efforts to protect the public health."

Key Points: 
  • "The FDA will continue our important work in this area as part of our efforts to protect the public health."
  • Zevtera's efficacy in treating SAB was evaluated in a randomized, controlled, double-blind, multinational, multicenter trial .
  • In the trial, researchers randomly assigned 390 subjects to receive Zevtera (192 subjects) or daptomycin plus optional aztreonam [the comparator] (198 subjects).
  • A total of 69.8% of subjects who received Zevtera achieved overall success compared to 68.7% of subjects who received the comparator.

Andelyn Biosciences and Grace Science, LLC Partner to Tech Transfer Phase I/II/III Manufacturing of a Suspension Process Adeno-Associated Virus (AAV) Gene Therapy for NGLY1 Deficiency

Retrieved on: 
Monday, March 25, 2024
ODD, AAV, CDMO, Disease, Deficit spending, Partnership, Grace, NGLY1, Patient, EMA, Hope, Priority review, NGLY1 deficiency, DSM-IV codes, Clinical trial, Marketing, FDA, Pharmaceutical industry

COLUMBUS, Ohio, March 25, 2024 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has partnered with Grace Science, LLC, to tech transfer and manufacture GS-100, a suspension process AAV NGLY1 gene therapy for Phase I/II/III clinical trial material for the treatment of NGLY1 Deficiency.

Key Points: 
  • COLUMBUS, Ohio, March 25, 2024 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has partnered with Grace Science, LLC, to tech transfer and manufacture GS-100, a suspension process AAV NGLY1 gene therapy for Phase I/II/III clinical trial material for the treatment of NGLY1 Deficiency.
  • The partnership will seek to maximize program efficiency, support the ongoing clinical trial, and accelerate this critical therapy to patients.
  • The partnership instills a quality mindset while also accelerating Grace's manufacturing timelines, offering hope for patients suffering from NGLY1 Deficiency.
  • Matt Wilsey, CEO and Co-Founder of Grace Science stated, "We are very excited about the partnership between Grace Science and Andelyn Biosciences.

FDA Approves First Treatment for Patients with Liver Scarring Due to Fatty Liver Disease

Retrieved on: 
Thursday, March 14, 2024
Research, Inflammation, Liver, Exercise, Disease, Drug, Dietary supplement, Patient, Fatty liver disease, Public, Animal, Immunology, Diarrhea, Marie Cassidy, Human services, Breakthrough therapy, Diet, Nausea, Priority review, Fibrosis, Heated tobacco product, Senior counsel, Safety, NASH, Hepatitis, Cosmetics, Vaccine, Food, Multimedia, Scar, FDA

NASH is a result of the progression of nonalcoholic fatty liver disease where liver inflammation, over time, can lead to liver scarring and liver dysfunction.

Key Points: 
  • NASH is a result of the progression of nonalcoholic fatty liver disease where liver inflammation, over time, can lead to liver scarring and liver dysfunction.
  • Rezdiffra is a partial activator of a thyroid hormone receptor; activation of this receptor by Rezdiffra in the liver reduces liver fat accumulation.
  • To enroll in the trial, patients needed to have a liver biopsy showing inflammation due to NASH with moderate or advanced liver scarring.
  • Patients should stop using Rezdiffra if they develop signs or symptoms of worsening liver function while on Rezdiffra treatment.