The Medicines and Healthcare products Regulatory Agency (MHRA) has granted an extension to the licensing of cystic fibrosis drugs, bringing hope to young patients

The Medicines and Healthcare Products Regulatory Agency (MHRA) has granted an extension to the licensing of cystic fibrosis medicines, bringing renewed hope to families battling the condition.

In a historic decision that brings hope to young families battling cystic fibrosis,… Medicines and Healthcare Products Regulatory Agency The MHRA has expanded the licensing of cystic fibrosis drugs Kaftrio (ivacaftor, tezacaftor, elexacaftor) and Kalideco (ivacaftor) to include children aged two to five years. This approval, supported by compelling evidence from studies in older patients and rigorous clinical trials in children ages two to five years, represents a major breakthrough in the management of 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 tract. This persistent buildup can lead to recurrent lung infections, breathing difficulties, and complications in nutrient absorption, severely affecting the quality of life of children with this condition. Taking Cafetrio and Kalydeco together as long-term treatment offers a glimmer of hope for these young patients.

These drugs, 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, Cafetrio and Kalydeko facilitate the movement of chloride ions into and out of cells. Enhanced chloride transport plays a pivotal role in loosening mucus and reducing inflammation. improve lung function, Ultimately relieving the debilitating symptoms of cystic fibrosis.

Cafetrio and Kalydeco for children aged 2 to 5 years include sachets of granules that are easily mixed with soft foods and taken before or after a meal or snack containing fat. The recommended dose for children weighing less than 14 kg is one sachet of Kaftrio granules (containing 60 mg ivacaftor, 40 mg tezacaftor, and 80 mg elixacaftor) in the morning, followed by one sachet of Kalydeco granules (containing 59.5 mg ivacaftor) in the evening. , with a guaranteed time interval. At least 12 hours between doses. For children weighing 14 kg or more, the recommended dose is 1 sachet of Kaftrio granules (containing 75 mg ivacaftor, 50 mg tezacaftor, and 100 mg eleccaftor) in the morning, followed by 1 sachet of Kalydeco granules (containing 75 mg ivacaftor) In the evening, again maintaining it. 12 hour interval between doses.

While the benefits of Kaftrio and Kalideco are significant, 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 receive the best possible treatment options and can enjoy a significantly improved quality of life.

The approval of Kaftrio and Kalideco for children ages 2 to 5 years represents a huge step forward in the fight against cystic fibrosis. With continuous research and dedicated efforts of healthcare professionals, The future for young patients suffering from this difficult condition holds the promise and possibility of a healthier life. A more fulfilling life.

About cystic fibrosis

Cystic fibrosis (CF) is a genetic disorder that relentlessly attacks the lungs and other vital organs. The root of the disease lies in a defective gene that regulates the production of thick, sticky mucus, which relentlessly clogs the airways and sets the stage for a constellation of debilitating complications. These complications include:

  • Recurrent lung infections
  • Pneumonia
  • Bronchitis
  • Bronchiectasis
  • pancreatitis
  • Liver disease
  • Infertility

Cystic fibrosis is a lifelong companion and can significantly impair an individual’s quality of life. However, with the advent of advanced treatment strategies, individuals with cystic fibrosis can live long and fulfilling lives.

on Medicines and Healthcare Products Regulatory Agency (MHRA)

The Medicines and Healthcare Products Regulatory Agency (MHRA) is a UK government agency responsible for regulating medicines, medical devices and blood components used in blood transfusions. The MHRA is an executive agency of the Department of Health and Social Care.

The primary responsibilities of the MHRA are:

  • Ensure that medications 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 aims. These powers include the ability to:

  • Granting drug marketing licenses
  • Medical device licensing
  • Conduct inspections on manufacturing sites
  • Remember unsafe or ineffective medications 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 website and through other channels, such as publications and briefings.

The Medicines and Healthcare products Regulatory Authority (MHRA) is a world-leading regulator, committed to working with its international partners to ensure safe and effective medicines and medical devices are available to patients around the world.

Here are some 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 encouraging innovation in the healthcare sector.


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