Amicus Therapeutics has a deeply emotional and personal backstory, can you walk us through what prompted you to bring the biotech company to life?
Amicus was founded as a result of our personal struggle to find a treatment for Pompe disease and while trying to find the most suitable biotech name we thought that the Latin equivalent for "friend" describes our vision the best. We strive to be a fully patient focused company in the industry and the industry certainly needs so much more work. There are nearly 8,000 rare diseases that affect more than 500 million people around the world. Ever since the beginning, we felt we had a moral obligation to develop the highest quality therapies for people living with rare diseases, at the fairest price and broadly accessible. This statement was coined a decade before we ever had an approved product and we have remained faithful to it until this day.
How did treatments and technologies change since you first started in the biotech field two decades ago?
By the late 90s, when I entered this industry, we had made good progress in cancer research, HIV/AIDS, and cardiovascular disease, but concepts like gene editing did not exist until ten years ago. The pace of technological progress has really been breathtaking, and I am glad that there has also been a change in the view of the purpose of our companies.
When our kids were diagnosed with Pompe disease there was no sliver of hope for a possible treatment. Thankfully, we managed to find a small number of researchers with early ideas and technologies and within a couple of years, developed an enzyme replacement therapy that eventually saved our children's lives. Prior to treatment, their hearts were three times the normal size and within 12 weeks they went back to normal. However, the gains in muscle strength plateaued after a while, meaning that, unfortunately, there is no silver bullet cure that permanently solves the problem.
How do you reconcile stories like that with the criticism the pharma industry still often receives from society?
Some of the pharma industry’s bad reputation is warranted, but this is changing for the better. The nefarious marketing and sales practices of the early days are out, but, according to surveys, the pharma industry remains one of the least respected in the U.S., next to big oil, tobacco, and the federal government. This is indeed a paradox since we always fought to gain an increasing recognition of the moral purpose of our activity.
The attitude towards rare diseases has changed as well – are big pharma companies working alongside you and how big is the progress that has been made overall?
Recently, we have been starting to see big pharma companies working in the field of rare diseases and that is a very welcome advancement. In addition to these, technologies like gene therapies or small molecules are finally coming of age, the potential for gene editing being especially exciting. We are entering a golden age of medicine in human genetic diseases, and this will certainly unlock the secrets for treating much more prevalent disorders like Parkinson's or Alzheimer's.
Having dealt with a rare disease on a personal level, what is your main takeaway about going through such an unfair experience?
When we began our journey we went through confusion, fear, anger, and finally settled for determination to learn everything we could about the disease. When faced with this adversity, the actions that you take are a measure of your character. As someone of faith, I had to integrate the idea that nature is not cruel, it is just brutally random. Looking back, all of us can be proud of the advancements we made, and we can be hopeful for the progress to come. We are flawed human beings but trying to find the strength to move forward is important and,
At the end of the day, biotechnology is just a great big word that means hope.
