We are a biopharmaceutical company focused on the commercialization of orphan drugs for the treatment of rare diseases. These diseases are of very low prevalence but have a great impact on patients who suffer from them. This is why we seek to be a beacon of hope for those patients whose illnesses are not a priority for society.
To be the Biotechnology company that offers, in the Latin American markets, the best treatments for the patients who need them the most.
Our vision is, until 2025, to make life easier for patients around LATAM.
Ruconest® (Recombinant C1 Esterase Inhibitor / Conestat Alfa) is an innovative drug for the treatment of Hereditary Angioedema. Ruconest® is a freeze-dried powder ready for reconstitution.
Hunterase® (Idursulfase Beta) is an innovative drug for the treatment of Hunter Syndrome (MPSII). Hunterase® is a clear, transparent injection containing a clear or opalescent solution.
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Panama City, Panama.
RUCONEST® (conestat alfa) is a recombinant human C1-esterase inhibitor (rhC1INH) approved for the treatment of angioedema attacks in patients with hereditary angioedema (HAE) in the US, Israel, South Korea, the 28 EU countries plus Norway, Iceland and Liechtenstein.
What is HAE?
HAE is a rare genetic condition that affects about 1 in 30,000 people worldwide. Symptoms include episodes of swelling in the hands, feet, abdomen, face, and airways. Patients may suffer attacks of unbearable abdominal pain, nausea, and vomiting that are exacerbated by swelling of the intestinal wall. Inflammation of the airways (larynx) is particularly dangerous and can cause death by asphyxiation. Without treatment, seizures can last between 48 and 120 hours and can be fatal. More information about the condition can be found on the website of the International Association of HAE Patients at www.HAEi.org
C1 esterase inhibitor (C1INH) is a protein found naturally in the human body. It belongs to the class of serine protease inhibitors or snakes. It regulates various inflammatory pathways in the body by inhibiting certain proteins (proteases) that are part of the human immune system. The deficiency of the functional inhibitor of C1 causes excessive activation of the complement system and other immunological and haemostatic pathways, leading to attacks of angioedema. These attacks are characterized by acute and painful swelling of the soft tissues. Administration of C1 inhibitor protein can normalize low levels of C1 INH and stop angioedema attacks.
Patients with HAE have a functional C1INH deficiency as a result of a mutation in the C1 inhibitor gene. For them, Pharming's RUCONEST® provides a causal treatment with a well-established efficacy and safety profile. Our patented production and purification technology complies with GMP standards, resulting in large-scale, reproducible, high-quality products. RUCONEST® does not carry the risk of transmission of plasma related human infectious agents as it is not isolated from human blood sources.
- Hunterase is an injection into a clear, colorless bottle containing a clear, slightly whitish, colorless solution. It is being developed for the second time in the world as a treatment for Hunter syndrome.
The recommended dose of Hunterase is 0.5 mg/kg body weight administered weekly as an intravenous infusion
. Hunterase is a concentrated solution for intravenous infusion and should be diluted in 100 mL of 0.9% sodium chloride. Each vial of Hunterase contains a 2.0 mg/mL solution of idursulfase - β protein (6.0 mg) in a 3.0 mL extractable volume and is for single use only. The use of an infusion set equipped with a 0.2 micron filter is recommended (μm).
The total volume of the infusion can be administered for a period of 1 to 3 hours. Patients may require longer infusion times due to infusion reactions; however, infusion times should not exceed 8 hours. The initial infusion dose should be 8 ml/hour for the first 15 minutes. If the infusion is well tolerated, the dose may be increased in increments of 8 mL/hour at 15-minute intervals to deliver the full volume within the desired time period. However, at no time should the rate of infusion exceed 100 mL/hour. The infusion rate may be slow and/or temporarily stopped, or administration may be stopped, based on clinical judgment, if reactions to the infusion occur. Hunterase should not be infused with other products in the infusion tube.
2. PREPARATION AND ADMINISTRATION INSTRUCTIONS: USE Hunterase
TECHNIQUES should be prepared and administered by a health professional.
Determine the total volume of Hunterase to be administered and the number of vials needed based on the patient's weight and the recommended dose of 0.5 mg/kg.
Patient Weight (kg) x 0.5 mg/kg Hunterase 2 mg/mL = Total # (mL) Hunterase
Total # (mL) Hunterase Total # (mL) Hunterase 3 mL/vial = Total # of (vial)
(Round up to determine the number of whole vials needed to extract the calculated volume of Hunterase to be administered.)
Visually inspect each vial. Hunterase is a clear solution to slightly whitish, colorless. Do not use if the solution in the vials is discolored or if particles are present. Hunterase must not be shaken.
Dilute the calculated total volume of Hunterase in 100 mL of 0.9% sodium chloride. After dilution, the solution in the infusion bag should be mixed gently, but not shaken. Diluted solution should be discarded if not administered or refrigerated within 8 hours of preparation. Diluted solution can be stored refrigerated for up to 48 hours.
Hunterase is for single use only. The rest of Hunterase after use should be discarded immediately.