Binghamton University faculty-created drug could ease Duchenne muscular dystrophy symptoms

Thirteen years of work by two leaders in Binghamton University's School of Pharmacy produced a drug designed to ease the symptoms of Duchenne muscular dystrophy while being free of commonly seen side effects.

That drug, Agamree (vamorolone), has now been approved by the Food and Drug Administration.

School of Pharmacy dean Kanneboyina Nagaraju and associate dean of research and research development Eric Hoffman developed vamorolone to treat patients 2 or older with DMD, a genetic condition that causes progressive muscle degeneration and shortened life expectancy.

What is DMD?

The genetic mutation causing DMD, found on the X chromosome, interferes with the production of dystrophin protein in muscle tissue, which is crucial to the growth and maintenance of muscles.

According to Nagaraju, only a few patients live past their 20s, even when treated with previously existing medicine.

"At the age of 2 to 4, normal kids jump around," said Nagaraju. "Kids who don't have dystrophin have to use their hands to get up from the floor. Almost all of these children, in their early teens, are wheelchair bound. Almost all of them die in their 20s."

Existing drugs, such as Prednisone, lessened symptoms and delayed their onset. The problem, according to Nagaraju, is that the drug causes a host of severe side effects. In order to lessen these side effects, doctors lower the dosage, which leads to DMD symptoms increasing.

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How did vamorolone receive FDA approval?

Nagaraju said his collaboration with Hoffman, who hired him in 2006, was critical in the development of vamorolone. Companies were more interested in larger, more famous diseases, Nagaraju said, and unwilling to fund research on diseases such as DMD.

"For me, suffering, whether it is a famous disease or a rare disease, is the same," said Nagaraju. "That has been our life's goal — to help these children."

Hoffman has worked on identifying and treating DMD for decades, beginning his work on DMD in 1985. Prior to vamorolone, Hoffman helped develop Viltolarsen, a muscular dystrophy drug which was FDA approved under an accelerated pathway. Nagaraju and Hoffman have been working to develop vamorolone for 13 years.

The process to earn full FDA approval involves large-scale clinical trials.

In a statement, Binghamton University said a double-blind placebo-controlled trial involving 121 patients — all 4- to 7-year-old boys — showed vamorolone increased strength and mobility. The trial was carried out across 32 academic hospitals and 11 countries.

According to Hoffman, patients with DMD can show decline in muscle functions and strength in just six months. When treated with vamorolone, patients' conditions improved.

"We used five different approaches looking at muscle functions, and all five showed significant improvement," said Hoffman. "First over six months, which is all the FDA asks for, but then we carried that on for a year, and many of those continued to show improvements."

Vamorolone, according to Nagaraju, has the same positive effects, with none of the negative side effects of existing drugs.

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What is next for vamorolone?

Nagaraju said the drug may also have other applications in the future, including asthma, allergies and other issues which drugs such as Prednisone are currently used for.

The European Medicines Association (EMA) has indicated they plan to make vamorolone the first drug to receive full approval in DMD treatment in Europe.

Hoffman said there are around 250 patients currently using vamorolone.

After trials end, patients are offered the option to continue using the drug — in the case of vamorolone, nearly every patient from various trials spanning 7 years has continued taking the drug.

Some trials are currently ongoing, with plans for more in the future.

This article originally appeared on Binghamton Press & Sun-Bulletin: Binghamton University professors see breakthrough in DMD treatment