Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules, biologics, and gene therapies that are in early to late-stage development.
Our Portfolio
The following chart summarizes our main product candidate portfolio.
IN-LICENSED PROGRAMS
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ACTIVATED PI3Kδ SYNDROME (APDS)
Activated Phosphoinositide 3-kinase Delta (PI3Kδ) Syndrome, or APDS is a rare, genetic, and progressive primary immunodeficiency. Discovered in 2013, APDS is a disorder that impairs the immune system and the function of the white blood cells that recognize and attack viruses and bacteria to prevent infection.
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OTL-105 – HSC GENE THERAPY FOR HAE
In 2021, Pharming announced a strategic collaboration with Orchard Therapeutics to research, develop, manufacture, and commercialize OTL-105, a newly disclosed investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of hereditary angioedema (HAE).
Hereditary angioedema, otherwise known as HAE, is a rare, serious, debilitating, potentially life-threatening genetic disorder occurring in 1 in 50,000 to 1 in 150,000 people worldwide. In Europe, approximately 10,000 people are affected. 1
HAE causes repeated episodes of spontaneous angioedema – or swelling – that can occur at any time and in almost any part of the body but more predominantly in the hands, feet, face, airway (throat) and internal organs, with airway or laryngeal, swelling being particularly dangerous and can lead to death by asphyxiation.
1. Ghazi A, Grant JA. Hereditary angioedema: epidemiology, management, and role of icatibant. Biol Targets Ther 2013;7:103. doi:10.2147 / BTT.S27566
ADDITIONAL PLATFORM PROGRAM
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POMPE
Pompe disease, also known as Acid Maltase Deficiency or Glycogen Storage Disease type II, is an inherited muscular myopathy disorder caused by the build-up of a polymer sugar called glycogen in the body’s cells. It’s a rare multisystem genetic disorder that is characterized by absence or deficiency of the lysosomal enzyme alpha-glucosidase, or GAA. This enzyme is required to break down, or metabolize, the complex carbohydrate glycogen and convert it into the simple sugar glucose. Failure to achieve its proper breakdown results in massive accumulation of lysosomal glycogen in cells, particularly in cardiac, smooth, and skeletal muscle cells.
rhC1INH FRANCHISE (DISCONTINUED)
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ACUTE KIDNEY INJURY
Acute kidney injury (AKI), also known as acute renal failure (ARF), is a sudden episode of kidney failure or kidney damage.
In August 2022, Pharming announced that we would discontinue further development of rhC1INH.
For more information, please read the press release outlining this decision.
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PRE-ECLAMPSIA
Pre-eclampsia is a life-threatening multisystem disorder in pregnancies leading to maternal and neonatal mortality and morbidity, usually first detected by hypertension.
In August 2022, Pharming announced that we would discontinue further development of rhC1INH.
For more information, please read the press release outlining this decision.