APDS

Pharming Group reports first quarter 2024 financial results and provides business update

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Mercoledì, Maggio 8, 2024
VWAP, VUS, FDA, Phase, Haploinsufficiency, IRP, Iberian languages, Laboratory, Safety, Euronext Amsterdam, Pharmacokinetics, PTEN, Epidemiology, CTLA4, Marketing, World Games, APDS, Health care, MAA, CHMP, Trial of the century, Genetic testing, Disease, Patient, Physician, EMA, Primary immunodeficiency, Ministry, Variant, Committee, MHRA, Diagnosis, Pids, PID, Lymphocyte, Clinical trial, Government, Microsoft Access, Prevalence, Ageing, Pharmaceutical industry

“Pharming delivered a strong first quarter, increasing quarterly revenues by 31% year-over-year to US$55.6 million and keeping the Company on track for our 2024 total revenue guidance of US$280 - US$295 million.

Key Points: 
  • “Pharming delivered a strong first quarter, increasing quarterly revenues by 31% year-over-year to US$55.6 million and keeping the Company on track for our 2024 total revenue guidance of US$280 - US$295 million.
  • Pharming made continued progress in the first quarter of 2024 on leniolisib regulatory filings for APDS patients 12 years of age and older in key global markets.
  • Pharming filed regulatory submissions in Canada and Australia in the third quarter of 2023, and Israel in the second quarter.
  • Pharming announced completion of enrollment in the clinical trial for children ages 4 to 11 years old on April 8, 2024.

Genomenon Partners with Pharming to Advance APDS Diagnosis

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Giovedì, Maggio 2, 2024
Mortality, American College, Probability, Laboratory, ANN, Survival, APDS, Disorder, Partnership, Genetic testing, PIK3R1, Molecular diagnostics, Disease, ACMG, Lymphoma, Patient, Bronchiectasis, AMP, Primary immunodeficiency, Caregiver, Genome, Immunodeficiency, Diagnosis, Infection, PIK3CD, Medicine, Activated, Medical genetics, Gene, Medical device

ANN ARBOR, Mich., May 2, 2024 /PRNewswire-PRWeb/ -- Genomenon, a genomic intelligence company, has partnered with Pharming Group in an effort to make variant data on the PIK3CD and PIK3R1 genes available to genetic testing labs, as well as clinicians and researchers, worldwide. This data enables accurate molecular diagnoses and supports the precise medical management of the disease. The partnership also improves the ability of diagnostic laboratories and clinicians to resolve variants of uncertain significance (VUSs) and advances Genomenon's mission to curate the human genome.

Key Points: 
  • APDS, or Activated PI3K delta syndrome, is a rare primary immunodeficiency affecting approximately 1 to 2 people per million worldwide.
  • As the symptoms of APDS can be associated with a variety of conditions, patients typically experience a median 7-year diagnostic delay.
  • "Through our AI-driven technology and genomic expertise, Genomenon has produced the world's most comprehensive variant landscape on PIK3CD and PIK3R1.
  • This curated genetic dataset is being made available to the community through Genomenon's Mastermind Genomic Intelligence Platform to help inform and accelerate the diagnosis for patients with APDS," said Mike Klein, Genomenon CEO.

Pharming to be honored as Industry Innovator at National Organization for Rare Disorders (NORD®) 2024 Rare Impact Awards

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Mercoledì, Aprile 24, 2024
FDA, Recognition, Food, Pediatrics, APDS, Patient, Infection, Immunity, Euronext Amsterdam, Organization, Autoimmunity

The Rare Impact Awards honor individuals, groups and organizations who are making exceptional strides to improve the lives of people living with rare diseases.

Key Points: 
  • The Rare Impact Awards honor individuals, groups and organizations who are making exceptional strides to improve the lives of people living with rare diseases.
  • Pharming will accept the award in recognition of the U.S. Food and Drug Administration (FDA) approval and commercialization of Joenja® (leniolisib), an oral, selective PI3Kδ inhibitor.
  • On behalf of the approximately 500 individuals in the US with this rare condition, NORD is thrilled to see such innovation taking place at Pharming.
  • With 95% of rare diseases not having therapies available, the approval of Joenja® for APDS is worthy of the NORD Industry Innovation Award.

National Organization for Rare Disorders (NORD) Announces 2024 Rare Impact Award Honorees, Emmy Award-Winning Journalist, Peter Alexander to Host

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Lunedì, Aprile 22, 2024
Education, FDA, Haemophilia, Risk, Genetics, Policy, Legislation, Louisiana House of Representatives, Diagnosis, Community, Enzyme replacement therapy, DSM-IV codes, Universal Pictures, Usher syndrome, NBC News, Primary immunodeficiency, Metabolic disorder, Therapy, Abbey, Coagulopathy, IgA nephropathy, Hospital, Rare disease, BioMarin Pharmaceutical, Proteinuria, Alpha-mannosidosis, Regeneron Pharmaceuticals, House, Immunoglobulin A, Medicine, Biogen, Universal Studios Hollywood, HFSC, PDT, APDS, Friedreich's ataxia, Disease, Science, Patient, Caregiver, Representative, Activated, Pharmaceutical industry

"Through science and advocacy, they are transforming the lives of those impacted by rare diseases, paving the way for new possibilities in treatment and care."

Key Points: 
  • "Through science and advocacy, they are transforming the lives of those impacted by rare diseases, paving the way for new possibilities in treatment and care."
  • "Having witnessed my sister's journey, I know the challenges rare disease families face are not just physical but also emotional, mental, and financial.
  • In addition to the individual award recipients, the Rare Impact Awards are also honoring companies that have developed treatments changing the lives of those with rare diseases.
  • Regeneron Pharmaceuticals for Veopoz™: The first and only treatment indicated specifically for CHAPLE disease, a rare hereditary immune disease.

Pharming announces completion of enrollment in pediatric clinical trial of leniolisib

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Lunedì, Aprile 8, 2024
Disease, Ageing, Patient, Euronext Amsterdam, Clinical trial, MPH, Index, Safety, FDA, APDS, Food, Pharmaceutical industry

Pharming plans to include data from this 4–11-year-old trial in regulatory filings worldwide for the approval of leniolisib for pediatric patients with APDS, beginning in 2025.

Key Points: 
  • Pharming plans to include data from this 4–11-year-old trial in regulatory filings worldwide for the approval of leniolisib for pediatric patients with APDS, beginning in 2025.
  • Anurag Relan, MD, MPH, Chief Medical Officer of Pharming, commented:
    “This is the first clinical trial for younger pediatric patients with APDS, who have a significant unmet need for a disease modifying treatment.
  • I am pleased with the progress made in our Phase III pediatric clinical program evaluating leniolisib in children with APDS.
  • 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.

Pharming Group reports fourth quarter and full year 2023 financial results

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Giovedì, Marzo 14, 2024
Patient, Health care, Epidemiology, Executive Committee, Laboratory, Microsoft Access, Lymphocyte, Clinical trial, European Commission, APDS, MHLW, AEG, Welfare, Ageing, Committee, EMA, Trial of the century, PID, CTLA4, Safety, Pharmacokinetics, Ministry, CHMP, ODD, U.S. FDA, Prevalence, PTEN, Disease, HAE, Immunodeficiency, VUS, Literature, PIK3R1, PMDA, FDA, DSM-IV codes, Diagnosis, Civil service commission, IRP, Haploinsufficiency, Genetic testing, MAA, Autoimmunity, World Games, Iberian languages, Marketing, MHRA, Euronext Amsterdam, PIK3CD, Physician, Pharmaceutical industry

The U.S. market contributed 97% of 2023 revenues, while the EU and Rest of World contributed 3%.

Key Points: 
  • The U.S. market contributed 97% of 2023 revenues, while the EU and Rest of World contributed 3%.
  • Revenues increased to US$7.9 million in the fourth quarter of 2023, driven by the continued increase in patients on paid therapy, and revenues were US$18.2 million for 2023.
  • Pharming made continued progress in the fourth quarter of 2023 on leniolisib regulatory filings for APDS patients 12 years of age and older in key global markets.
  • Pharming filed regulatory submissions in Canada and Australia in the third quarter of 2023, and Israel in the second quarter.

Pharming Group announces updated full year 2023 guidance and Joenja® U.S. launch update

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Lunedì, Gennaio 8, 2024
APDS, PIK3CD, VUS, FDA, Disease, Immunodeficiency, PIK3R1, Laboratory, Euronext Amsterdam, Growth, Patient, Pids, HAE, Pharmaceutical industry

For the full year 2023, total revenues are expected to increase by 19% to approximately US$245 million (preliminary and unaudited*).

Key Points: 
  • For the full year 2023, total revenues are expected to increase by 19% to approximately US$245 million (preliminary and unaudited*).
  • We expect 10% growth in RUCONEST® revenues, significantly exceeding our previous guidance for low single digit annual revenue growth.
  • In 2023, Pharming saw the first full-year, post-pandemic benefit of its first quarter 2020 restructuring and expansion of its U.S. salesforce.
  • Pharming expects to issue full financial results for the fourth quarter and full year 2023 in March 2024.

Pharming Group announces development plans for leniolisib for additional primary immunodeficiencies (PIDs)

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Mercoledì, Dicembre 13, 2023
APDS, Food, FRCPA, Infection, NIAID, IND, Epidemiology, FDA, Prevalence, Immunodeficiency, Autoimmune lymphoproliferative syndrome, CTLA4, Pharmacokinetics, Primary immunodeficiency, Euronext Amsterdam, Protocol, Lymphocyte, PTEN, Gene, Bangladesh Technical Education Board, Face, NIH, Patient, Safety, FAS, PID, Pharmaceutical industry

Leiden, The Netherlands, December 13, 2023: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces today the expansion of its rare disease pipeline with plans to develop leniolisib for additional primary immunodeficiencies (PIDs) beyond activated phosphoinositide 3-kinase delta syndrome (APDS).

Key Points: 
  • Leiden, The Netherlands, December 13, 2023: Pharming Group N.V. (“Pharming” or “the Company”) (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces today the expansion of its rare disease pipeline with plans to develop leniolisib for additional primary immunodeficiencies (PIDs) beyond activated phosphoinositide 3-kinase delta syndrome (APDS).
  • Pharming has engaged with the US Food and Drug Administration (FDA) and has received feedback on its plans to develop leniolisib for PID disorders with immune dysregulation.
  • The Phase 2 clinical trial will evaluate leniolisib in PIDs with immune dysregulation linked to PI3Kẟ signaling in lymphocytes, with similar clinical phenotypes to APDS.
  • Building upon the success of leniolisib for APDS, we believe that leniolisib will continue to have efficient uses in rebalancing immune dysregulation in PIDs.

Pharming announces first patient dosed in pediatric clinical trial for children aged 1 to 6 years for leniolisib

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Martedì, Novembre 21, 2023
Associate, APDS, Food, FDA, Hope, Ageing, Rheumatology, MPH, Diagnosis, Safety, Patient, University, Pharmaceutical industry

At sites in the U.S., Japan, and the EU, the single-arm, open-label, multinational clinical trial will evaluate the safety, tolerability, and efficacy of leniolisib in 15 children, one to six years of age, who have a confirmed APDS diagnosis.

Key Points: 
  • At sites in the U.S., Japan, and the EU, the single-arm, open-label, multinational clinical trial will evaluate the safety, tolerability, and efficacy of leniolisib in 15 children, one to six years of age, who have a confirmed APDS diagnosis.
  • These patients will receive a specific, pediatric granulated formulation of leniolisib.
  • The study’s primary efficacy endpoints and secondary endpoints mirror those used to evaluate the clinical outcomes in previous leniolisib Phase II/III APDS trials for patients aged 12 and older.
  • While standard supportive therapies can ease some of the progressive, immune-related symptoms that characterize APDS, leniolisib is designed to help prevent those symptoms from arising.

APDS Announces New Name: Orijin™

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Lunedì, Novembre 13, 2023
APDS, Employment, Curriculum

NEW YORK, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Today, American Prison Data Systems (APDS), announces a major renaming effort to become Orijin.

Key Points: 
  • NEW YORK, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Today, American Prison Data Systems (APDS), announces a major renaming effort to become Orijin.
  • Like APDS, Orijin’s mission is to prepare every justice-impacted individual for sustainable employment.
  • “American Prison Data Systems (APDS) was founded in 2012.
  • Nearly ten years later, we’re still focused on ‘changing corrections for good’ but revitalizing our dedication and work with a new name called Orijin,” stated Orijin’s Co-Founder Arti Finn.