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What is Acthar?
This new 'old' anti-inflammatory medication has ophthalmic applications.
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Welcome to another edition of CEDARS/ASPENS Debates. CEDARS/ASPENS is a joint society of cornea, cataract and refractive surgery specialists, here to discuss some of the latest hot topics in ophthalmology.
In recent years, there have been numerous new medications evaluated for treatment of ocular inflammation. As with all new medications, there has been the issue of cost. This month, Melissa Toyos, MD, FACS, discusses a controversial new “old” anti-inflammatory medication that, while it may have tremendous potential to treat many conditions, may be cost prohibitive. We hope you enjoy the discussion.
Kenneth A. Beckman, MD, FACSOSN CEDARS/ASPENS Debates Editor
H.P. Acthar gel is one of the newest old medications making an impact in ophthalmology. Owned by Mallinckrodt Pharmaceuticals, Acthar has a new sales force in ophthalmology and a wide open-label indication for all severe acute and chronic allergic and inflammatory processes involving the eye and its adnexa, including but not limited to keratitis, iritis, iridocyclitis, diffuse posterior uveitis and choroiditis, optic neuritis, chorioretinitis, and anterior segment inflammation.
Acthar is an adrenocorticotropic hormone (ACTH) that is thought to quell inflammation by stimulating the body’s own ability to produce endogenous steroids as well as affecting steroid-independent immunomodulatory and anti-inflammatory pathways through the binding of melanocorticotropin receptors. Melanocorticotropin receptors are found on a range of immune cells — T helper cells, T regulatory cells, dendritic cells and macrophages — as well as on retinal pigment cells.
Like most hormones, the original form of ACTH had a half-life of only 10 minutes, so Acthar was developed for clinical use by creating a repository gel that can be injected intramuscularly or subcutaneously and tailored according to the needs of the patient. H.P. Acthar gel comes in 5 mL vials containing 80 USP units per milliliter. Most commonly patients receive 0.5 mL to 1 mL injections one to three times weekly.
Side effects associated with Acthar are similar to those of steroids and include blood pressure and blood sugar derangements, symptom masking, susceptibility to new infections, Cushingoid symptoms, GI bleeding, loss of bone density, weight gain, mood swings, cataracts and glaucoma. Patients started on Acthar therapy should be monitored by their primary care physician for common side effects.
Acthar has made the news for other reasons. Acthar was the subject of a 60 Minutes story on rising drug prices. Rising drug prices are a serious issue facing our country and our patients. In Acthar’s case, it was first introduced to medicine in 1952 and had approval at that time to treat all steroid-responsive conditions. A true orphan drug, Sanofi ultimately decided to discontinue the drug because of lack of profit. Pediatric neurologists who felt Acthar was essential for treatment of infantile spasms appealed strongly to the FDA for a label and cost review in order to ensure its continued availability for their patients. Since acquiring Acthar, Mallinckrodt has made only modest price adjustments. Each vial is currently priced at $40,000.
Acthar has been most commonly used and studied in multiple sclerosis, infantile spasms, nephrology and rheumatology. Apart from some published case reports on cicatricial pemphigoid and optic neuritis, little published data currently exist on the use of Acthar in ophthalmology.
Mallinckrodt is actively supporting clinical research into the drug and has invested nearly $400 million into that goal. As of the time of this writing, there were more than 300 studies registered with ClinicalTrials.gov looking at uveitis, dry eye, retinal vasculitis, optic neuritis and inflammatory conditions unresponsive to methylprednisolone. In our clinic, we have utilized Acthar for some of our recalcitrant scleritis and dry eye patients. Two scleritis patients who received Acthar were incompletely controlled with maximal topical steroids and nonsteroidals, and both experienced uncontrolled steroid-responsive glaucoma. In both cases, within 3 to 6 weeks of beginning Acthar therapy, patients were weaned off all topical medications, including topical glaucoma medications. Response in dry eye has been variable, and we are currently undertaking an investigator-initiated study to look into this more closely.
Acthar is a new “old” option for acute and chronic allergy and inflammation, especially in patients unresponsive to traditional therapies or intolerant of synthetic steroids. We have much to learn about where and how Acthar may be most useful in ophthalmology, and only clinical experience and well-organized trials can answer these important questions.
- References:
- ClinicalTrials.gov.
- Gettig J, et al. P T. 2009;34(5):250-257.
- H.P. Acthar Gel. Information for healthcare professionals. www.acthar.com/healthcare-professional. Accessed May 16, 2018.
- Mallinckrodt shares rebound after ‘60 Minutes’ report on drug prices. www.cnbc.com/2018/05/07/mallinckrodt-shares-rebound-after-60-minutes-report-on-drug-prices.html. Updated May 7, 2018.
- Questcor — The deal of a lifetime. seekingalpha.com/article/2061773-questcor-the-deal-of-a-lifetime. Published March 3, 2014.
- For more information:
- Melissa Toyos, MD, FACS, can be reached at Toyos Clinic, 2204 Crestmoor Road, Nashville, TN 37215; email: mtoyos@toyosclinic.com.
Disclosure: Toyos reports she is a consultant for Mallinckrodt and is currently receiving a grant for investigator-initiated research.