Updated March 27, 2019
Our mission at Mallinckrodt is Managing Complexity. Improving Lives. Our employees live this mission every day, and we're focused on providing safe, effective treatments that make a difference in the lives of patients, especially those with severe and critical conditions.
A number of media outlets and other external parties have made misleading and inaccurate allegations about Acthar Gel (repository corticotropin injection) and Mallinckrodt. It is important to set the record straight.
Here are key facts:
Please see additional facts below along with other resources.
March 27, 2019
Acthar Gel (repository corticotropin injection) is for many frequently, very sick patients, a life-changing drug. We are proud of Acthar Gel and the important investment we are making in it, and we are gratified that we can positively impact patients' lives through this drug.
Acthar Gel is FDA-Approved for 19 Indications
While Acthar Gel has been used to treat patients for more than 50 years, its label was reviewed by the FDA in 2010, at which time the FDA determined there was sufficient scientific and clinical evidence to support its use in the 19 various indications1 contained in the current Acthar Gel label.
One of these indications is for use of Acthar Gel in the treatment of Infantile Spasms (IS)2, for which it is considered the gold-standard for treatment. Two randomized clinical trials were submitted in support of FDA approval of the drug and its effectiveness as a treatment for IS, one of which compared Acthar Gel to prednisone. In that trial 86.7% of patients had a positive response to Acthar Gel vs. 28.6% that responded to prednisone. Patients who responded in the pivotal study treated with a two-week course of Acthar Gel therapy experienced complete suppression of the two key measures of disease – spasms and hypsarrhythmia. The IS clinical trial results appear in Section 14 of the full prescribing information for the drug.
Aside from treatment of IS, Acthar Gel is often prescribed by doctors predominantly as a later-line treatment to a small subset of patients suffering from various devastating diseases for whom other approved FDA treatment options have failed.
The Price of Acthar Gel
In 2007, Acthar Gel’s previous owner was near bankruptcy and raised the price of the drug substantially in order to keep the drug on the market and to ensure the long-term supply of the drug for treatment of children afflicted with infantile spasms and other small groups of patients suffering from complex, devastating diseases. They did this only after extensive consultation with the FDA.3
Today the price per vial of Acthar Gel is $38,892. Since acquiring Acthar Gel, Mallinckrodt has only made modest price adjustments in the mid-single digit percentage range. Additionally, Mallinckrodt provides discounts to this list price to payers, which the prior owner generally did not offer. We would encourage you to review Mallinckrodt's pledge on drug pricing and innovation, which we take very seriously.
Availability of Acthar Gel for Infantile Spasms Patients
In short, Mallinckrodt invests significant resources to provide Acthar to babies quickly and with the utmost urgency. There is an entire support team that passionately and personally ushers each baby’s prescription through the process to ensure no delays.
NEW: Mallinckrodt’s Investment in Acthar Gel
Since acquiring Acthar Gel in 2014, Mallinckrodt has invested more than $500 million into the drug, specifically: building on substantial clinical experience as well as previously completed and largely independent clinical case series and smaller trials; modernizing manufacturing; expanding medical affairs and research activities; and initiating seven well-designed, company-sponsored randomized, controlled clinical studies, targeting combined enrollment of nearly 1,100 patients.3
Significant Clinical Evidence Supports the Efficacy of Acthar Gel
There is significant clinical evidence to support the effectiveness of Acthar Gel. This evidence is the result of company-sponsored controlled clinical trials, investigator-initiated research conducted in top hospitals and medical centers by some of the country's preeminent physicians, and health economic and outcomes research data. Equally important, there are decades of clinical experience that doctors have with the product as a proven therapy for appropriate patients.
As an FDA-approved drug, Acthar Gel is deemed safe and effective for its labeled indications by the agency. Since acquiring Acthar Gel, Mallinckrodt has, though, continued to conduct post-approval clinical studies in a number of key indications. Along with the wealth of clinical experience gained over the decades with this drug, this data will assist physicians in the use of Acthar Gel in the most appropriate patient populations.
NEW: On February 5, 2019, Mallinckrodt provided a data update on its Phase 4 Acthar Gel clinical trial in patients with rheumatoid arthritis and reported the open-label phase of the study was completed. Learn more in the News section.
Physician Payments/Travel Expense Reimbursement:
In the period of 2013-2016, of all healthcare practitioners prescribing Acthar Gel to whom Mallinckrodt or the prior owner made payments, more than 95% received only modest meals or nominally priced clinical reprints – well within regulations and guidelines. For the remaining ~5%, the vast majority were engaged for peer-to-peer speaking engagements, with a small fraction involved in other consulting services for the company, such as speaking to employees or investors and participation in expert Physician Advisory Boards – again, all within regulations and guidelines. It is our belief that many physicians prefer peer-to-peer presentations and dialogue over other methods of learning about the value a product may bring to appropriate patients they are treating. The physicians who present to their peers must take time away from their practice and frequently travel to other cities – incurring normal, but sometimes substantial travel expenses. Any payments reported include reimbursement for these expenses.
Mallinckrodt designs our policies to be consistent with applicable legal and regulatory requirements, the PhRMA Code, and industry best practices. We have instituted controls and strict guidelines regarding the selection and training of speakers; the conduct of such programs, including requirements related to the individuals that may attend such programs; and guidelines to ensure that the venues selected for such programs are appropriate and conducive to the educational focus of these programs, and payments are based on fair market value.
As an FDA-approved drug, Acthar Gel is deemed safe and effective for its labeled indications by the agency.
Mallinckrodt annually provides the FDA with data about adverse events related to its marketed products. As we approach our yearly filing, we are pleased to report that the positive benefit-risk of Acthar Gel has remained unchanged across all marketed indications and is consistent with previous years. To derive meaningful conclusions of this topic, adverse event reports for Acthar Gel need to be considered within the appropriate context.
Acthar Gel is typically prescribed to patients with very serious medical conditions, often as a third or fourth line of treatment when other treatments have failed. It is well known that many of these patients suffer from diseases in which co-morbidities are high, and often they are on other medications that may be contributing factors. The frequency of adverse event reports also does not necessarily correlate to an increase in the actual prevalence or relative severity of any particular side effect or event. Each event is reported and counted whether it relates to a relatively minor event such as a headache or a more serious event such as anaphylaxis.
Furthermore, the FDA itself cautions on its website that reporting of a side effect or adverse event occurring while taking a drug doesn’t establish a causal relationship between the adverse event and the medicine.
Over the past years, the number of patients using Acthar Gel has increased significantly. Critically, however, company-generated Acthar Gel data on adverse events over the last four calendar years indicates that the number of serious adverse events as a proportion of the number of Acthar Gel prescriptions (measured by vials sold) has remained very low and consistent with the FDA’s independent analysis.
Acthar Gel Advisory Committee Results
Regarding your questions on this topic, these are the facts: On May 11, 2010, a meeting of the FDA’s Peripheral and Central Nervous System Drugs Division held an Advisory Committee Meeting (AdCom) to review/discuss the data Questcor was submitting/had submitted in support of use of Acthar Gel in treatment of patients with Infantile Spasms. The Committee voted 22 to 1 that Acthar Gel was an effective treatment for patients with IS and voted 20 to 1 the drug was safe in the intended patient population at an effective dosing regimen, inclusive of a Risk Evaluation and Mitigation Strategy (REMS). The REMS was a part of the agency’s eventual approval of the drug for this indication (along with the reaffirmation of 18 others) in 2010. Two years later, the FDA removed the REMS requirement based on Acthar Gel’s demonstrated safety in the market in IS patients.
The Facts about the Rockford Lawsuit
Mallinckrodt strongly believes that none of the company actions outlined in the plaintiff’s complaint constitute a violation of any law and, therefore, believes that the complaint should be dismissed in its entirety. We will vigorously defend the company in this matter.
Treating physicians prescribe what they believe is best for their patients and the doctor(s) in Rockford, Illinois chose to prescribe Acthar Gel. The medical community is well aware that there are other treatment options for IS such as high dose steroids.
Synacthen, Acthar Gel and the FTC Settlement
NEW: Synacthen is not a generic competitor to Acthar Gel. While the two drugs may share mechanistic effects through the ACTH component, Acthar Gel is much more. As noted in the product label approved by the FDA, Acthar Gel is a naturally-sourced complex mixture of purified adrenocorticotropic hormone analogs and other pituitary peptides. It is not a steroid, and amongst its many components includes a 1-39 peptide chain. It is not simply a highly purified form of ACTH. Synacthen is a synthetic ACTH 24-peptide chain. The two products are very different drugs.
Mallinckrodt did not pursue commercialization of Synacthen for IS, as the barriers to completion were, in our view, virtually impossible to overcome.
Mallinckrodt is developing the drug (MNK-1411) in an indication where there is both high unmet medical need and, if successful, potential for greatest impact for patients – Duchenne Muscular Dystrophy.
Thank you for taking the time to read this important information.
1The FDA conducted a thorough review of the label for Acthar Gel in 2010.
2Because symptoms of IS can be subtle and are generally not widely recognized, Mallinckrodt invests resources to support education of the medical and patient community to ensure IS babies are getting diagnosed promptly. We also invest in ongoing clinical research to further understand the disease and since treating infantile spasms is so urgent once diagnosed Mallinckrodt has established an entire support team to usher each baby’s prescription through the coverage process to ensure quick access to the product.
3Please see the Questcor’s 10-K filing for 2007. Additionally, Questcor issued many new shares the year before, presumably to raise capital. Moreover, articles have appeared, including one in Investor’s Business Daily in November of 2013 in which the former CEO of Questcor publicly discussed the company’s challenges to stay afloat during this time period.
4Patients who withdrew from the process on their own would be exceptions to this statement.
5The seven ongoing clinical trials are referenced below. Each link takes you to details about the study that can be found on www.clinicaltrials.gov. These important investments will build upon the existing body of clinical evidence to support the effectiveness of Acthar Gel.
• Phase 2B trial in Amyotrophic Lateral Sclerosis
• NEW: Phase 4 trial in Uveitis
• Phase 4 trial in Pulmonary Sarcoidosis
• Phase 4 trial in Multiple Sclerosis Relapse
• Phase 4 trial in Lupus
• Phase 4 trial in Rheumatoid Arthritis
• Phase 4 trial in Focal Segmental Glomerulosclerosis (Nephrotic Syndrome)
About Acthar Gel (repository corticotropin injection)
Acthar Gel is an injectable drug approved by the FDA for the treatment of 19 indications. Of these, today the majority of Acthar use is in these indications:
IMPORTANT SAFETY INFORMATION
Warnings and Precautions
Other adverse events reported are included in the full Prescribing Information.
Please see full Prescribing Information available at Acthar.com.
Each of the stories below provide a view of patients who are taking action to treat their disease or condition with Acthar.