New drug delivery technology for “Cystic Fibrosis”

Contents

Drug delivery system

Cystic fibrosis

Ivacaftor

Traditional dosage form of “IVACAFTOR”

Proposed dosage form

Advantages of the proposed dosage form

Side-effect reduction of proposed dosage form

Conclusion

Drug delivery system

Refers to the technology which is utilized to present the

drug to the desired body site for drug release &

absorption.

Cystic fibrosis

• A progressive & genetic disease that causes persistent lung

infections and limits the ability to breathe over time. It

may also affects the pancreas, liver, kidneys, and intestine.

Drugs used to treat cystic fibrosis

Ivacaftor

• Formula : C24H28N2O3

• Molecular Weight : 392.490 g/mol

• Generic name : Ivacaftor.

• Brand name : Kalydeco.

• Drug approved : Jan 31,2012.

• Cystic fibrosis usage approved : May 17,2017.

Traditional dosage form of “IVACAFTOR”

Tablet

Film-coated

Oral Granules.

Proposed dosage form

Buccal Tablet.

• A topical route of administration by which drugs are

applied in the cheek at canine fossa through the diffusion

of oral mucosa which enters directly into the bloodstream.

Advantages of the proposed dosage form

1. Better Bio-availability : Traditional dosage forms of

“Ivacaftor” has 99% protein binding. So bio-availability is

almost lowest.

2. Rapid onset of action : Tmax is around 3-5 hours. So it

takes more time to show it’s therapeutic effect.

3. Avoid first-pass metabolism : By liver due to direct entry

into the bloodstream.

Side-effect reduction of proposed dosage form

1. Soar throat : Pain or irritation of the throat caused by

inflammation & trauma. (Local)

2. Sneezing : Uncontrollable expulsion of air through the

nose & mouth. (Local)

3. Runny nose : Production of extra mucus by the nose.

(Local)

Side-effect reduction of traditional dosage form

4. Nasal congestion : Blockage of the nasal passages

usually due to inflamed blood vessels. (Local)

5. Loss of appetite.

Conclusion

• “Ivacaftor” was sold for $339M in USA during the year of

2014 for other indications. As it’s usage for cystic fibrosis is

approved by US-FDA recently, it has huge opportunity to

do well in this disease condition too. Also if we can

successfully formulate the buccal form of “Ivacaftor”, then

it will boost the usages & sales of “Ivacaftor” further

more.

References

• 1. https://en.wikipedia.org/wiki/Cystic_fibrosis

• 2. https://www.cff.org/What-is-CF/About-Cystic-Fibrosis/

• 3. http://www.deltagene.eu/cftr

• 4. https://en.wikipedia.org/wiki/Ivacaftor

• 5. https://en.wikipedia.org/wiki/First_pass_effect

• 6. https://www.drugs.com/kalydeco.html

• 7. https://www.drugs.com/sfx/kalydeco-side-effects.html

• 8. https://www.drugs.com/dosage/kalydeco.html