PASLI disease

PANTHERx® Rare Launches Drug for APDS

Retrieved on: 
Tuesday, March 28, 2023
Urinary tract infection, PIK3CD, Failure, Attention deficit hyperactivity disorder predominantly inattentive, Pharmacy, Immunodeficiency, Quality of life, Mutation, Medicine, Sinusitis, PI3K, PASLI disease, Patient, Bronchiectasis, Primary immunodeficiency, Rare, PIK3R1, Headache, Gene, APDS, Pharmaceutical industry, Vaccine

Through innovation and technology, we continue to make a positive impact on the quality of life of those living with rare diseases.

Key Points: 
  • Through innovation and technology, we continue to make a positive impact on the quality of life of those living with rare diseases.
  • We are pleased to add Joenja® to the list of medications in PANTHERx® portfolio," said Rob Snyder, president of PANTHERx® Rare.
  • Activated Phosphoinositide 3-Kinase-Delta Syndrome, or APDS, is a rare primary immunodeficiency affecting approximately one to two people per million with disease onset occurring as early as infancy.
  • Those with APDS may develop recurrent respiratory tract infections, bronchiectasis, lymphoproliferation as well as neurodevelopment delay and failure to thrive.

Pharming announces US FDA approval of Joenja® (leniolisib) as the first and only treatment indicated for APDS

Retrieved on: 
Friday, March 24, 2023
US Foods, PIK3CD, Fetus, Senior counsel, Birth, New Drug Application, Atopic dermatitis, Autoimmunity, Associate, Committee, Incidence, MD, Patient, Risk, Ageing, Disease, Priority review, Hope, EMA, Primary immunodeficiency, Diagnosis, FDA, Headache, Caregiver, Safety, Rheumatology, Liver, PASLI disease, Marketing Authorisation Application, Lymph, University, European Medicines Agency, MAA, PIK3R1, Splenomegaly, CHMP, Lymphoma, ANC, PRV, Euronext Amsterdam, Infection, Clinical trial, NDA, Sale, Sinusitis, Physician, Immunodeficiency, Genetic testing, Gene, Food, APDS, Pharmaceutical industry, Management, Vaccine, Pharming, Novartis, Medicine

LEIDEN, The Netherlands, March 24, 2023 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that the US Food and Drug Administration (FDA) has approved Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency. The FDA evaluated the Joenja® application for APDS under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. Joenja® is expected to launch in the US in early April and will be available for shipment in mid-April.

Key Points: 
  • Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency.
  • The FDA approval of a treatment option for one of the more than 450 primary immunodeficiencies is also a key moment for the broader primary immunodeficiency community.
  • Until now, management of APDS has relied on the treatment of the diverse symptoms associated with APDS.
  • With the approval of Joenja®, as a treatment for a rare pediatric disease, the FDA granted Pharming a priority review voucher ("PRV").

Pharming announces US FDA approval of Joenja® (leniolisib) as the first and only treatment indicated for APDS

Retrieved on: 
Friday, March 24, 2023
US Foods, PIK3CD, Fetus, Senior counsel, Birth, New Drug Application, Atopic dermatitis, Autoimmunity, Associate, Committee, Incidence, MD, Patient, Risk, Ageing, Disease, Priority review, Hope, EMA, Primary immunodeficiency, Diagnosis, FDA, Headache, Caregiver, Safety, Rheumatology, Liver, PASLI disease, Marketing Authorisation Application, Lymph, University, European Medicines Agency, MAA, PIK3R1, Splenomegaly, CHMP, Lymphoma, ANC, PRV, Euronext Amsterdam, Infection, Clinical trial, NDA, Sale, Sinusitis, Physician, Immunodeficiency, Genetic testing, Gene, Food, APDS, Pharmaceutical industry, Management, Vaccine, Pharming, Novartis, Medicine

LEIDEN, The Netherlands, March 24, 2023 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that the US Food and Drug Administration (FDA) has approved Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency. The FDA evaluated the Joenja® application for APDS under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. Joenja® is expected to launch in the US in early April and will be available for shipment in mid-April.

Key Points: 
  • Joenja®, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency.
  • The FDA approval of a treatment option for one of the more than 450 primary immunodeficiencies is also a key moment for the broader primary immunodeficiency community.
  • Until now, management of APDS has relied on the treatment of the diverse symptoms associated with APDS.
  • With the approval of Joenja®, as a treatment for a rare pediatric disease, the FDA granted Pharming a priority review voucher ("PRV").

Pharming announces first patient enrolled in pediatric clinical trial of leniolisib

Retrieved on: 
Tuesday, February 21, 2023
APDS, New Drug Application, American Society of Hematology, Rheumatology, Ageing, CHMP, MAA, Diagnosis, MHRA, PDUFA, Safety, Division, Patient, HIV disease progression rates, Committee, Clinical trial, PASLI disease, EMA, Food, Committee for Medicinal Products for Human Use, European Medicines Agency, Index, Health care, Disease, FDA, MPH, Phase, Society, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, European Directive on Traditional Herbal Medicinal Products, Prescription Drug User Fee Act, PIP, UCLA, Marketing Authorisation Application, Doctor of Philosophy, Vaccine, Pharmaceutical industry, Pharming, MD

LEIDEN, Netherlands, Feb. 21, 2023 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM)/(Nasdaq: PHAR) announces that the first patient has been enrolled in its Phase III clinical trial (NCT05438407) evaluating the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, in children with activated phosphoinositide 3-kinase delta syndrome (APDS). There is currently no approved treatment for this complex and progressive disease caused by genetic variants.

Key Points: 
  • Pharming plans to initiate a similar clinical trial in the third quarter of 2023 that will include children aged 1 to 6 years, with APDS, to evaluate a new pediatric formulation of leniolisib.
  • Eligible patients enrolled in either of the pediatric trials will continue to receive leniolisib for a year after the initial 12-week treatment period through an open-label extension trial.
  • Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:
    "I am pleased we have initiated the first trial in our Phase III pediatric clinical program evaluating leniolisib in children with APDS, and I look forward to our second pediatric trial getting underway.
  • Pharming expects that the CHMP will issue its opinion on the leniolisib MAA in 2H 2023.

Pharming announces first patient enrolled in pediatric clinical trial of leniolisib

Retrieved on: 
Tuesday, February 21, 2023
APDS, New Drug Application, American Society of Hematology, Rheumatology, Ageing, CHMP, MAA, Diagnosis, MHRA, PDUFA, Safety, Division, Patient, HIV disease progression rates, Committee, Clinical trial, PASLI disease, EMA, Food, Committee for Medicinal Products for Human Use, European Medicines Agency, Index, Health care, Disease, FDA, MPH, Phase, Society, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, European Directive on Traditional Herbal Medicinal Products, Prescription Drug User Fee Act, PIP, UCLA, Marketing Authorisation Application, Doctor of Philosophy, Vaccine, Pharmaceutical industry, Pharming, MD

LEIDEN, Netherlands, Feb. 21, 2023 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (EURONEXT Amsterdam: PHARM)/(Nasdaq: PHAR) announces that the first patient has been enrolled in its Phase III clinical trial (NCT05438407) evaluating the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, in children with activated phosphoinositide 3-kinase delta syndrome (APDS). There is currently no approved treatment for this complex and progressive disease caused by genetic variants.

Key Points: 
  • Pharming plans to initiate a similar clinical trial in the third quarter of 2023 that will include children aged 1 to 6 years, with APDS, to evaluate a new pediatric formulation of leniolisib.
  • Eligible patients enrolled in either of the pediatric trials will continue to receive leniolisib for a year after the initial 12-week treatment period through an open-label extension trial.
  • Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:
    "I am pleased we have initiated the first trial in our Phase III pediatric clinical program evaluating leniolisib in children with APDS, and I look forward to our second pediatric trial getting underway.
  • Pharming expects that the CHMP will issue its opinion on the leniolisib MAA in 2H 2023.