MHRA Grants License Extension for Cystic Fibrosis Medications, Bringing Hope to Young Patients

MHRA grants License extension for cystic fibrosis medications, bringing renewed hope to families battling the condition.

In a landmark decision that brings hope to young families battling cystic fibrosis, the Medicines and Healthcare products Regulatory Agency (MHRA) has extended the license of the cystic fibrosis medicines Kaftrio (ivacaftor, tezacaftor, and elexacaftor) and Kalydeco (ivacaftor) to include children aged two to five years old. This approval, backed by compelling evidence from studies in older patients and a rigorous clinical trial in children aged two to five, represents a significant breakthrough in managing the debilitating symptoms of cystic fibrosis in this vulnerable age group.

Cystic fibrosis, an inherited genetic disorder, is characterized by the accumulation of thick, sticky mucus in the lungs and digestive system. This relentless buildup can lead to recurrent lung infections, breathing difficulties, and complications with nutrient absorption, severely impacting the quality of life for children with this condition. Kaftrio and Kalydeco, administered together as a long-term treatment, offer a beacon of hope for these young patients.

These medicines, specifically designed to target patients with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene – the most common genetic mutation associated with cystic fibrosis – work by interacting with defective CFTR proteins. By encouraging these proteins to open more frequently, Kaftrio and Kalydeco facilitate the movement of chloride ions in and out of cells. This enhanced chloride transport plays a pivotal role in thinning mucus, reducing inflammation, and improving lung function, ultimately alleviating the debilitating symptoms of cystic fibrosis.

The administration of Kaftrio and Kalydeco for children aged two to five years involves sachets of granules that are conveniently mixed with soft food and consumed just before or after a meal or snack containing fat. The recommended dosage for children weighing under 14kg is one sachet of Kaftrio granules (containing 60mg ivacaftor, 40mg tezacaftor, and 80mg elexacaftor) in the morning, followed by one sachet of Kalydeco granules (containing 59.5mg ivacaftor) in the evening, ensuring an interval of at least 12 hours between doses. For children weighing 14kg or more, the recommended dosage is one sachet of Kaftrio granules (containing 75mg ivacaftor, 50mg tezacaftor, and 100mg elexacaftor) in the morning, followed by one sachet of Kalydeco granules (containing 75mg ivacaftor) in the evening, again maintaining a 12-hour interval between doses.

While the benefits of Kaftrio and Kalydeco are substantial, the MHRA remains steadfast in its commitment to ongoing monitoring of the safety and effectiveness of these medicines in this younger age group. This commitment reflects the agency’s unwavering dedication to ensuring that children with cystic fibrosis have access to the best possible treatment options and can experience a significantly improved quality of life.

The approval of Kaftrio and Kalydeco for children aged two to five years marks a monumental step forward in the fight against cystic fibrosis. With continued research and dedicated efforts from healthcare professionals, the future for young patients living with this challenging condition holds promise and the prospect of a healthier, more fulfilling life.

About Cystic Fibrosis

Cystic fibrosis (CF) is a genetic disorder that relentlessly assaults the lungs and other vital organs. Its genesis lies in a defective gene that orchestrates the production of a thick, sticky mucus, relentlessly clogging the airways and paving the way for a constellation of debilitating complications. These complications include:

  • Frequent lung infections
  • Pneumonia
  • Bronchitis
  • Bronchiectasis
  • Pancreatitis
  • Liver disease
  • Infertility

CF is a lifelong companion that can significantly impair one’s quality of life. However, with the advent of sophisticated treatment strategies, individuals with CF can lead long and fulfilling lives.

About the Medicines and Healthcare products Regulatory Agency (MHRA)

The Medicines and Healthcare products Regulatory Agency (MHRA) is a government agency in the United Kingdom that is responsible for regulating medicines, medical devices, and blood components for transfusion. The MHRA is an executive agency of the Department of Health and Social Care.

The MHRA’s primary responsibilities are to:

  • Ensure that medicines and medical devices are safe and effective for use
  • Protect public health
  • Promote innovation

The MHRA has a wide range of powers to achieve these objectives. These powers include the ability to:

  • Grant authorizations for the marketing of medicines
  • License medical devices
  • Conduct inspections of manufacturing sites
  • Recall unsafe or ineffective medicines and medical devices

The MHRA is also responsible for providing information to the public about medicines and medical devices. This information is available on the MHRA’s website and through other channels, such as publications and briefings.

The MHRA is a world-leading regulator and is committed to working with its international partners to ensure that safe and effective medicines and medical devices are available to patients around the world.

Here are some of the key things to know about the MHRA:

  • The MHRA is independent of the pharmaceutical industry.
  • The MHRA’s decisions are based on scientific evidence.
  • The MHRA is committed to transparency and accountability.

The MHRA plays a vital role in protecting public health and promoting innovation in the healthcare sector.

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