FDA Has Declined to Approve New Medications
Senator, Congressman Call on FDA To Answer Questions About Approval Process
WASHINGTON, DC – Today, U.S. Senator Bob Casey (D-PA) and Congressman Todd Platts (R-PA) demanded answers from the Food and Drug Administration (FDA) as Pennsylvania families struggle to access lifesaving medications.
The FDA has declined to approve new treatments for Fabry disease and amyloidosis even though the drugs in question are available in other countries. The agency’s lack of approval of the new drugs has impacted Pennsylvania families who were hoping these potentially lifesaving drugs would provide new and better treatment options. In a letter to the FDA’s Commissioner, Margaret Hamburg, Senator Casey and Congressman Platts asked for a detailed explanation of the agency’s decision.
They wrote, “In the wake of the lack of FDA approval of both these NDAs, we have received many inquiries from concerned patients and their families about why the FDA did not approve the treatments. In particular, since both products have been approved by the European Medicines Agency (the European regulatory equivalent to the FDA) and are available to patients in Europe, there are a lot of questions about why the FDA would not approve these products for use by patients in the United States.”
Fabry disease, a genetic condition, is estimated to affect 1 in 40,000-60,000 people. The condition results from abnormal deposits of a particular fatty substance throughout the body. Over time, this buildup can lead to life-threatening complications such as progressive kidney damage, heart attack, and stroke. Similarly, amyloidosis is a group of diseases that result from the abnormal deposition of a particular protein, called amyloid, in various tissues of the body. Familial amyloidosis, with an estimated incidence of less than 1 per 100,000, often affects nerves and kidneys and can lead to failure of the kidneys, other major organs, and death.
Recently, the FDA considered two new drug applications (NDAs) that were separately submitted for treatment of each of these serious diseases. While patients in both communities were hopeful that the FDA would approve these NDAs, unfortunately, neither was approved; one – the Fabry product called Replagal – was withdrawn by the application sponsor due to concerns that FDA was leaning toward not approving the product without further clinical trials. The withdrawal and lack of approval of what had been viewed as very promising drug candidates has been devastating to patients and their families, who had been holding out hope for clinical advancements in treatments for these life-threatening illnesses for which there is little currently available to help patients manage, let alone cure.
Although rare, Fabry affects residents across Pennsylvania. York resident Bill Shorb said, “Both my wife, Mari, and son, Patrick, are affected by Fabry disease and it has been devastating to my family. Despite responding well to Replagal, they have had to switch back to a less effective treatment that has suffered from numerous production problems, leading to a drug shortage and some very bad outcomes for my family and the Fabry community at large. Replagal represented a new and promising option for my family that has now been taken away.”
Shorb continued, “We need to get serious about approving viable options for patients suffering from rare diseases. My wife and son have suffered from mini-strokes (TIA’s), double vision, heart conditions, and more. I just want my wife and son to be able to get the medications they need to live a good life.”
In the case of familial amyloidosis, there is currently no FDA-approved treatment available to patients. While there is one FDA-approved product available to those with Fabry disease, the company that holds a patent and produces has not been able to reliably produce enough product historically to meet the needs of the entire United States market.
The full text of the letter Senator Casey and Congressman Platts sent to the FDA is below:
The Honorable Margaret Hamburg
Commissioner
Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993
Dear Commissioner Hamburg:
We write to request your assistance on behalf of Pennsylvania patients and their families who have contacted our offices because of serious problems they face accessing appropriate Food and Drug Administration (FDA)-approved medications to treat their life-threatening conditions.
Over the past year, we have heard from numerous patients and their families in Pennsylvania affected by two lethal diseases: Fabry disease and familial amyloidosis. Both of these diseases are rare and inherited. Fabry disease, which is estimated to affect 1 in 40,000-60,000 males (and fewer females), results from abnormal deposits of a particular fatty substance throughout the body. Over time, this buildup can lead to life-threatening complications such as progressive kidney damage, heart attack, and stroke. Similarly, amyloidosis is a group of diseases that result from the abnormal deposition of a particular protein, called amyloid, in various tissues of the body. Familial amyloidosis, with an estimated incidence of less than 1 per 100,000, often affects nerves and kidneys and can lead to failure of the kidneys, other major organs, and death.
Recently, the FDA considered two new drug applications (NDAs) that were separately submitted for treatment of each of these serious diseases. While patients in both communities were hopeful that the FDA would approve these NDAs, unfortunately, neither was approved; one – the Fabry product – was withdrawn by the application sponsor due to concerns that FDA was leaning toward not approving the product without further clinical trials. The withdrawal and lack of approval of what had been viewed as very promising drug candidates has been devastating to patients and their families, who had been holding out hope for clinical advancements in treatments for these life-threatening illnesses for which there is little currently available to help patients manage, let alone cure, them.
In the case of familial amyloidosis, there is currently no FDA-approved treatment available to patients. While there is one FDA-approved product available to those with Fabry disease, the company that holds a patent and produces it has not been able to reliably produce enough product historically to meet the needs of the entire United States market. Further, many of the Fabry patients who were given access to the product under consideration in the NDA through a compassionate use program responded more positively to it than the FDA-approved product, according to their and their doctors’ testimonials. Therefore, it would be beneficial to have both Fabry products available to patients in the United States not just to prevent shortages, but also due to clinical benefit associated with unique patient needs and responses.
In the wake of the lack of FDA approval of both these NDAs, we have received many inquiries from concerned patients and their families about why the FDA did not approve the treatments. In particular, since both products have been approved by the European Medicines Agency (the European regulatory equivalent to the FDA) and are available to patients in Europe, there are a lot of questions about why the FDA would not approve these products for use by patients in the United States This is particularly confusing to the Fabry community, where the product has been available in Europe for more than a decade and there appear to be no known safety concerns.
Frankly, we share their concerns and their questions, and would ask that you provide us with responses to the following questions to help us, and these patients, better understand the FDA’s process for approving new drug applications for rare diseases for which there are few, or no, alternatives:
• How did the FDA evaluate the safety and efficacy data provided in each NDA, and consider the risks and benefits of the specific needs of patients, especially in light of the fact that both the NDAs were for rare diseases that often lead to death and which few, if any, reliable treatment options exist?
• What consideration was given to the approval of these drugs in Europe, and, in the case of the Fabry product approved ten years ago, record of safe and effective use in Europe and in 43 countries around the world?
• In the case of rare diseases, what type of consideration is given to utilizing observational data and post-marketing trials (which may be conducted outside the U.S. at the request of a foreign regulatory agency) to assess the safety and efficacy of a drug?
• Under what circumstances does the FDA require a sponsor to conduct further clinical studies to secure approval?
• How does the FDA take into consideration the unique, and often acute, health care needs of United States patients with rare diseases in its calculations about the relative risks and benefits of the drug in a manner that allows it to “strike the right balance” with respect to its decision to approve or deny approval of a drug?
• What options exist for patients residing in the United States to receive these two products through either a Compassionate Use exemption or through personal importation?
Thank you for your consideration of this request.
Sincerely,
Robert P. Casey, Jr.
United States Senator
Todd Russell Platts
United States Representative
###
