IG Therapy

FDA Approves First Proven Treatment for Dermatomyositis

Until recently, those who live with dermatomyositis (DM), have relied on off-label medications to treat this rare autoimmune disease of the muscles. On July 16, however, the Food and Drug Administration (FDA) approved an intravenous immune globulin (IVIG) therapy called Octagam10% for use by adults with DM.

“This is fantastic news for the myositis community,” says Dr. Rohit Aggarwal. “Octagam is the first proven FDA approved treatment for a myositis indication. We’ve had drugs in the past, but this is the first one that has the kind of scientific evidence—a Phase 3 clinical trial that is randomized, double blind, and placebo controlled—that is required for FDA approval.”

Dr. Aggarwal, an internationally recognized myositis expert, was the principal investigator for the Phase 3 clinical trial called ProDERM that demonstrated that Octagam 10% caused significant improvement in DM skin and muscle symptoms as well as other disease criteria.

Immune globulin is a concentrated solution of antibodies derived from donated human plasma. While immune globulin has been used to treat a variety of autoimmune diseases for more than 30 years, it’s use in myositis has been “off-label.” In fact, with a few grandfathered-in exceptions, all treatments for myositis diseases are used without an FDA labeled indication (approval) for this condition.

For a rare disease like myositis, however, it’s often difficult to convince a pharmaceutical company to invest in the research necessary to attain FDA approval. Fortunately, Octapharma, a privately owned pharma company based in Switzerland, chose to take that leap with Octagam.

“We are very responsive to patient and clinician requests,” says Eric Pluckhorn, US director of sales for Octapharma. “This now gives us an entree into the autoimmune market that up until now we really have not had for the Octagam brand.”

Several years ago when the company started this project, DM represented a huge unmet need. At the time, Dr. Aggarwal and his colleagues had recently developed response criteria that would provide the kind of measurable data needed to clearly demonstrate the drug’s benefit for patients. The success of the trial is good news for both DM patients and the providers who care for them.

For patients, off-label use often makes accessing the medication a challenge. IVIG is very effective, but it’s also very expensive. Health insurance companies, in an effort to cut costs, often don’t want to pay for such costly treatments. So they create roadblocks for patients, such as requiring them to first try a number of other treatments without success before they allow IVIG. This practice is called “step therapy.” If the drug is prescribed off-label, many companies simply refuse to pay for it.

Practitioners are also enthusiastic. Empirically,we always knew IVIG works,” Dr. Aggarwal says, “but we were hesitant to give it, partly because of not having Phase 3 clinical trial-level of evidence and partly due to the insurance issues. Now we have an approved drug that we can give to the patients, and insurance companies can no longer deny it because it’s off-label.”

Perhaps more importantly, Dr. Aggarwal suggests this approval sets a precedent for future drug development in myositis.

“Once you have a drug approved through the FDA based on a rigorous process and a valid set of criteria, other drug companies will look at that as a pathway for novel drug clinical trials in myositis,” he says. “The ProDERM study sets a precedence for future drug approval in myositis.”

Within the next five to ten years, Dr. Aggarwal predicts there will be a number of novel therapies approved for myositis diseases, including not only dermatomyositis, but also polymyositis and necrotizing myopathy.

To address the other side of the access equation—that is, the expense—Octapharma plans to offer financial assistance to help cover the cost of the treatment. Eligible patients with commercial health insurance will have access to the company’s Copay Assistance Program that will cover out-of-pocket costs, such as coinsurance, copay expenses, and deductibles, up to $2,500 per year. This assistance is provided without regard for ability to pay. (By law in the US, the company is not able to offer this coverage to those who have government plans such as Medicare, Medicaid, or Tricare.) Patients who don’t have insurance or have lost their insurance may also get assistance through a compassionate use program.

“The FDA approval of Octagam 10% as a safe, tolerable and efficacious treatment for dermatomyositis in adults is exciting news for patients who previously relied on unapproved treatments,” said Octapharma USA President Flemming Nielsen. “Octapharma is committed to providing life-saving and life-enhancing therapies for patients with rare diseases. We look forward to partnering with patient organizations and the medical community to develop educational and other support programs that will serve dermatomyositis patients.”

Patient communities

Connecting with Patients is the Reward

As a research scientist, immunologist Huub Kreuwel, PhD never really worked with patients. He spent most of his time in an academic lab, trying to understand the basic biology of certain diseases and identifying molecules that could serve as targets for new therapies. He never got to see what happened in the later stages of drug development—that part where patients got better because of the discoveries he’d made.

When he left academia to serve as medical science liaison at Johnson and Johnson, however, he discovered a whole new experience. Now, years later, as Vice President for Scientific and Medical Affairs in the United States for Octapharma, talking to patients and providers about the plasma-based products his company produces is the best part of his job.

“When I came out of academia, I found it was very satisfying to actually talk to a patient who had tried our drug and had good results,” he says. “As an immunologist, it made sense to work on a lot of these rare diseases like primary immune deficiency and dermatomyositis. And it’s gotten more and more interesting over the years.”

Working in the medical affairs department also offers the opportunity to get involved with a wide variety of projects. Huub and his team work with regulatory agencies when the company is seeking approval for new products. They help set up clinical trials to test new therapies and answer physicians’ questions about how those therapies work. Best of all, he meets the people who benefit from Octapharma’s treatments, such as immune globulin (IG) therapies, and helps them enroll as research subjects in the company’s clinical trials.

Recently, the company completed a trial testing intravenous immune globulin (IVIG) therapy in patients with dermatomyositis (DM). While the results have not yet been made public, Huub says the trial did meet its primary endpoints, so it looks very promising that Octagam 10% will eventually become one of the few FDA-approved treatments for this disabling disease that affects the skin and muscles.

Part of what made this trial so successful was the feedback Huub and his team received from patients. In the process of developing the clinical trial, they worked with patient organizations, including The Myositis Association and Myositis Support and Understanding, to understand how patients experienced the disease so they could improve the study protocol and to help recruit participants for the trial.

“We work on a lot of orphan drugs,” Huub says. “And there aren’t that many patients sometimes, so we need everybody to help us to finish these trials. It worked quite well in the DM trial. Those were very productive relationships.”

The success Octapharma had with this phase III clinical trial with DM will also pave the way for future clinical trials for this indication. When rare diseases have few previous clinical trials, researchers often fumble to find tests that will tell them whether a particular drug is working or not. Octapharma’s trial in DM not only proved that the treatment was effective, it also showed that their measures of effectiveness worked in this patient population.

Huub is now developing protocols to test Octapharma products with other diseases. Among these are pediatric autoimmune neuropsychiatric disorders associated with streptococcal infections (PANDAS for short—a disease in which psychiatric symptoms such as obsessive-compulsive disorder appear suddenly after a strep infection) and secondary immune deficiency (SID—a problem that occurs when immune system deficiencies occur because of something other than genetics, such as HIV or chemotherapy).

As they did with the DM study, he and his team are talking to patients to get input that will improve these studies. One way they do this is by recruiting an advisory board of about a dozen patients who spend the day with company representatives sharing their experiences and suggestions. These open-ended discussions provide insights into all manner of ideas: how to better explain data, ideas for new trials, how patients need to be supported during a trial, and more.

“Those discussions are really good for the company, and usually they’re very productive,” Huub says. “Often patients have ideas for new products or practical solutions that might make our products better. And a lot of times it actually has led to either different products or different marketing material or revamping our website or providing patient education sessions.”

These days the thing that has captured Huub’s interest is COVID-19. Healthcare providers on the front lines of the pandemic are finding success in treating the virus with IG. In fact, recent events have made Octapharma a leader in exploring new therapies for COVID-19.

The company is currently supporting two investigator-initiated projects—one testing IVIG as a treatment for COVID-related respiratory failure, the other using IVIG and steroids to treat COVID-19 patients who are developing heart problems. Octapharma is also conducting their own phase III clinical trial to see if high-dose IVIG can be used to improve severe COVID-19 symptoms. Initial results from the investigator-initiated study with COVID-related respiratory failure are very promising.

“Of course COVID is horrible,” Huub says. “But it also became an opportunity for us to delve deeper into IVIG and how it can potentially work in that disease. It’s very satisfying for me personally and for my team to try and come up with other drugs that could help COVID. So overall, it’s been a very interesting ride.”