Pegaptanib
Intravitreal injection | |
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Pharmacokinetic data | |
Elimination half-life | 10 days |
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Chemical and physical data | |
Formula | C294H342F13N107Na28O188P28[C2H4O](m+n) (m+n≈900) |
Molar mass | ~50 kg/mol |
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Pegaptanib
Mechanism of action
Pegaptanib is a
Pegaptanib works as an
Means of administration
Pegaptanib is administered in a 0.3 mg dose once every six weeks by
Preclinical trials
Pegaptanib underwent several
Animal toxicology studies
Toxicology studies were conducted in
Clinical studies
Phase I
Phase I studies began in 1998 under Eyetech Pharmaceuticals. This study was conducted in 15 patients with wet AMD. Doses ranging from 0.25 to 30 mg per eye were injected into the eye, and patients were monitored for a period of three months. The results showed that 80% of the patients had stabilization or improvement, and 26.7% were showing improvement with no signs of toxicity.[6]
Phase II
After the success of the Phase I study, Eyetech completed a Phase II study focusing on multiple injections. In this study, 21 patients with subfoveal choroidal neovascularization (CNV) secondary to AMD were given multiple intravitreal injections. Due to the presence of subfoveal CNV some patients were given a secondary treatment, photodynamic therapy (PDT) for this condition. Results showed that in 87.5% of patients who received only pegaptanib, vision stabilized or improved. In patients who received PDT alone only 50.5% saw a slight improvement. However, when the two therapies were administered together, the level of improvement reached 60% or better.[6]
Phase III
After the success of the Phase I and Phase II trials, the FDA granted
The primary efficacy endpoint for the study was denoted by the proportion of the patients who lost less than 15 letters of visual acuity from their assessed baseline over the 54-week assessment.[5]
Results of the first year showcased promising results for pegaptanib. In groups treated with the 0.3 mg dose at the primary efficacy endpoint, experienced a statistically significant result.
- Study 1: 73% pegaptanib vs. 60% placebo
- Study 2: 67% pegaptanib vs. 53% placebo
Also, on average, pegaptanib 0.3 mg treated patients as well as the placebo patients continued to experience vision loss. However, the rate of vision decline was significantly lower than that of patients with the placebo treatment.[5]
Moreover, it was also determined that the second year of treatment was less effective than the first year. Results of the primary efficacy endpoint are:
- Study 1: 57% pegaptanib vs. 56% placebo
- Study 2: 61% pegaptanib vs. 34% placebo
Regulatory approval information
Pegaptanib (pegaptanib sodium injection) has been approved in: United States (2004)[7] Europe (2005)[3] Brazil (2005)[7] Canada (2006)[6]
Pending: Australia (Filed in 2006)
This section needs to be updated.(August 2016) |
Side effects
Common side effects of pegaptanib include:[3]
- Anterior chamberinflammation
- Raised intraocular pressure
- Punctate keratitis(small marks on the surface of the eye)
- Vitreous floaters (small particles or spots in the vision)
- Retinal damage (extremely adverse)
- Endophthalmitis (an infection inside the eye)
- Vitreous haemorrhage(bleeding inside of the eye)
Commercialization
The average cost of pegaptanib was approximately $5,300 per 5 syringes in the US. In 2004, when pegaptanib was approved it was a novel drug in its target and treatment for the treatment of AMD. However, the last large market sales occurred in 2010. Shortly after in 2011, sales began to decline due to the development of a more effective treatment, ranibizumab (a monoclonal antibody, Novartis) being developed and sold,[8] and the off-label use of the cheaper Bevacizumab.[9]
References
- ^ Drug Information: Pegaptanib Sodium Injection Archived 2013-12-14 at the Wayback Machine
- ^ "Pegaptanib". go.drugbank.com. Retrieved 2023-10-24.
- ^ a b c d e "Macugen (pegaptanib)" (PDF). European Medicines Agency: 1–3. 2010. Retrieved 2013-12-08.
- ^ "Larry Gold and Craig Tuerk (NeXstar Pharmaceuticals, Boulder, USA)". European Patent Office. 2011-02-16. Retrieved 2013-12-08.
- ^ a b c d "Highlights of Prescribing Information (Macugen)". Food and Drug Administration: 3–12. July 2007.
- ^ PMID 17717967.
- ^ a b "Eyetech Announces Approval of Macugen(R) in Brazil for the Treatment of Neovascular (Wet) Age-Related Macular Degeneration". Evaluate. 2005. Retrieved 2013-12-08.
- ^ "Macugen product information". Evaluate. 2011. Retrieved 2013-12-08.
- S2CID 8694305.