PIK3CD

Pharming Announces European Medicines Agency (EMA) Validates its Marketing Authorisation Application under Accelerated Assessment for leniolisib

Retrieved on: 
Friday, October 28, 2022
Gene, Phase, MAA, PIK3R1, U.S. Securities and Exchange Commission, Company, Attention deficit hyperactivity disorder predominantly inattentive, Primary immunodeficiency, Marketing, Safety, Disclosure, Forward-looking statement, Goal, B cell, PDK1, PIP2, AKT, PIK3CD, European Economic Area, Cell, Immunodeficiency, Committee, PI3K, European Medicines Agency, Macrophage, MPH, Growth hacking, Genetic testing, Metabolism, Annual report, Patient, Phrase, Immune system, APDS, Euronext Amsterdam, Committee for Medicinal Products for Human Use, Review, Autoimmunity, PIP3, CHMP, Time, Marketing Authorisation Application, Neutrophil, Advanced Therapy Medicinal Product, EMA, Public health, Vaccine, Pharmaceutical industry, MD, Pharming

LEIDEN, Netherlands, Oct. 28, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM / Nasdaq: PHAR) announces today that its Marketing Authorisation Application (MAA) for leniolisib has been validated for scientific evaluation under an accelerated assessment by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP). The application, submitted earlier in October 2022, is for the investigational drug, leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adolescents and adults 12 years or older.

Key Points: 
  • LEIDEN, Netherlands, Oct. 28, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM / Nasdaq: PHAR) announces today that its Marketing Authorisation Application (MAA) for leniolisib has been validated for scientific evaluation under an accelerated assessment by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use ( CHMP ).
  • In August 2022, Pharming announced the leniolisib MAA was granted accelerated assessment by EMA's CHMP.
  • Marketing authorisation for leniolisib in the European Economic Area is anticipated in H1 2023.
  • This review constitutes a key milestone in Pharming's effort to give healthcare providers and their patients global access to leniolisib.

Pharming Announces US FDA Acceptance for Priority Review of its New Drug Application for Leniolisib

Retrieved on: 
Wednesday, September 28, 2022
Attention deficit hyperactivity disorder predominantly inattentive, MPH, PDK1, B cell, Time, Safety, Lymphadenopathy, COVID-19, Autoimmunity, Risk, FDA, Food, Neutrophil, Phase, U.S. Securities and Exchange Commission, Gene, Population, Company, Degenerative disease, Macrophage, NDA, PDUFA, Lists of diseases, Metabolism, PIK3R1, Patient, PI3K, AKT, Cell, Euronext Amsterdam, Genetic testing, Annual report, Primary immunodeficiency, PIP3, Priority review, New Drug Application, APDS, NASDAQ, US Foods, Growth hacking, Index, Classification, Lymphoma, PIK3CD, Disease, Disclosure, Immunodeficiency, Pharmaceutical industry, Vaccine, MD, Pharming

LEIDEN, The Netherlands, Sept. 28, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (Euronext Amsterdam: PHARM) (NASDAQ: PHAR) announces that the US Food and Drug Administration (FDA) has accepted for priority review its New Drug Application (NDA) for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, to treat the rare primary immunodeficiency activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older in the US. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 29, 2023, aligned with a Priority Review classification.

Key Points: 
  • The FDA has assigned a Prescription Drug User Fee Act (PDUFA)goaldate of March 29, 2023, aligned with a Priority Review classification.
  • Furthermore, safety data from the study showed that leniolisib was well tolerated by participants.
  • Also submitted as part of the application were data from a long-term, open-label extension clinical trial including 38 patients with APDS who were treated with leniolisib for a median of 102 weeks.
  • Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:
    "The FDA's acceptance for priority review of Pharming's New Drug Application for leniolisib is a milestone that demonstrates our commitment to addressing unmet needs for patients with rare diseases.

Pharming Announces US FDA Acceptance for Priority Review of its New Drug Application for Leniolisib

Retrieved on: 
Wednesday, September 28, 2022
Attention deficit hyperactivity disorder predominantly inattentive, MPH, PDK1, B cell, Time, Safety, Lymphadenopathy, COVID-19, Autoimmunity, Risk, FDA, Food, Neutrophil, Phase, U.S. Securities and Exchange Commission, Gene, Population, Company, Degenerative disease, Macrophage, NDA, PDUFA, Lists of diseases, Metabolism, PIK3R1, Patient, PI3K, AKT, Cell, Euronext Amsterdam, Genetic testing, Annual report, Primary immunodeficiency, PIP3, Priority review, New Drug Application, APDS, NASDAQ, US Foods, Growth hacking, Index, Classification, Lymphoma, PIK3CD, Disease, Disclosure, Immunodeficiency, Pharmaceutical industry, Vaccine, MD, Pharming

LEIDEN, The Netherlands, Sept. 28, 2022 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (Euronext Amsterdam: PHARM) (NASDAQ: PHAR) announces that the US Food and Drug Administration (FDA) has accepted for priority review its New Drug Application (NDA) for leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, to treat the rare primary immunodeficiency activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older in the US. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of March 29, 2023, aligned with a Priority Review classification.

Key Points: 
  • The FDA has assigned a Prescription Drug User Fee Act (PDUFA)goaldate of March 29, 2023, aligned with a Priority Review classification.
  • Furthermore, safety data from the study showed that leniolisib was well tolerated by participants.
  • Also submitted as part of the application were data from a long-term, open-label extension clinical trial including 38 patients with APDS who were treated with leniolisib for a median of 102 weeks.
  • Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:
    "The FDA's acceptance for priority review of Pharming's New Drug Application for leniolisib is a milestone that demonstrates our commitment to addressing unmet needs for patients with rare diseases.