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News in Context: FDA "Breakthrough Therapy Designation" and Acadia's Nuplazid

News in Context: FDA "Breakthrough Therapy Designation" and Acadia's Nuplazid

Update Posted January 9, 2023: Many people and families with Parkinson’s disease (PD) worry about the possibility of developing hallucinations (seeing things that aren’t there) or delusions (believing things that aren’t true). These symptoms don’t happen in everyone, and they aren’t always bothersome. But for some, they can be disruptive or frightening. When that happens, doctors may recommend medication.

In 2016, the U.S. Food and Drug Administration approved Nuplazid (pimavanserin) to treat hallucinations and delusions in people with PD. Early on, questions were raised about the drug’s safety. But new research, including this publication, suggests the drug is as safe as other medications that treat psychosis. All anti-psychosis drugs, including Nuplazid, may in general, increase the risk of death in older people who have psychosis and significant cognitive changes (dementia). But it’s unclear whether this increased risk is because of the medication or the symptoms.

All medications have possible side effects and benefits. Always work with your doctor to determine which is the best option for you or your loved one.

The U.S. Food and Drug Administration (FDA) issued a statement on September 20, 2018 following its review of Nuplazid, a drug approved for Parkinson's psychosis (hallucinations and delusions). The agency wrote that it did not identify any new or unexpected safety findings and that it stood by its earlier decision that the drug's benefits outweigh the risks. 

"Patients taking Nuplazid for Parkinson’s disease psychosis should continue to use it as prescribed by their health care provider," wrote the FDA. Read the full report.

Update posted September 22, 2021: Top Parkinson’s physicians are questioning recent research, published in Neurology, on risks linked to the use of Nuplazid (pimavanserin). The study found that people with Parkinson’s over age 65 who were taking Nuplazid had a higher risk of hospitalization or death compared to people who were not taking the drug. However, the study had significant limitations, including that comparison groups were not equally matched. The authors compared people with Parkinson’s rather than people with Parkinson’s who had psychosis. Because people on Nuplazid had psychosis, and because psychosis is often associated with more advancing disease, which can increase risk for hospitalization and other complications, it’s not possible to determine whether differences were due to advancing Parkinson’s, the medication or other factors.

Clinical trials found Nuplazid to be safe and effective, and the FDA approved the medication in 2016. Learn more about the drug, and always talk with your personal physician about possible benefits and side effects when considering any medication.

Update posted June 7, 2018:  Neurology Today published a brief on June 7 regarding the FDA's re-examining of the antipsychotic drug Nuplazid (pimavanserin) as reported by CNN and other media outlets. The report notes the agency said, "Based on these data, the FDA has, at this time, not identified a specific safety issue that is not already adequately described in the product labelling." The brief also includes positive comments about Nuplazid from several Parkinson's experts. 


Originally posted April 10, 2018: On April 9, 2018, CNN published an article about the safety of Nuplazid, the first drug specifically indicated for the treatment of Parkinson's disease psychosis. Nuplazid (pimavanserin) was approved by the U.S. Food and Drug Administration (FDA) in April 2016 under the agency's Breakthrough Therapy Designation, one of four expedited review programs at FDA, applicable to drugs that have shown substantial improvement over existing treatments in early clinical data.

Breakthrough status is requested by the drug company and, if granted, makes companies eligible to receive additional guidance from the FDA on trial design, safety and efficacy standards, and pathways to approval. To receive breakthrough designation, drugmakers are required to show an effect on a serious or life-threatening condition or symptom. The FDA review process for these therapies may be expedited to more quickly provide treatment options for serious conditions and fill unmet medical needs.

As the nearly 1 million Americans living with Parkinson's can attest, new and better treatment options are urgently needed to sufficiently address the wide-ranging motor and non-motor symptoms of the disease. The process of translating early-stage findings into proven and practical treatments is, too often, slow and inefficient, including the years devoted to clinical testing and regulatory approval. Policies such as breakthrough designation have been created by Congress over the last quarter-century in an effort to get therapies to patients faster, while still upholding strict safety and efficacy standards.

The advent of the first Parkinson's disease psychosis treatment represents an important step in understanding and treating PD holistically. However, every drug brings potential risks and side effects.

As with every aspect of Parkinson's, if you or a loved one are living with Parkinson's psychosis, it is vital to work closely with your doctor (ideally a movement disorder specialist) to understand and evaluate the potential benefits and risks of any therapy. This includes seeking as much information as possible from your care provider before starting any drug, but also keeping lines of communication open as you integrate it into your regimen, and making note of any changes (positive or negative) you notice. Some patients and care partners report that they find it helpful to journal, and/or to ask close family and friends to share any observations of changes in behavior or symptoms, in order to keep an accurate record that can be shared at a doctor's appointment.

The Michael J. Fox Foundation will continue to monitor and report any new developments around the status of Nuplazid.

A note on our relationship with Acadia Pharmaceuticals

MJFF did not fund the development of Nuplazid, though our clinical trial search engine, Fox Trial Finder, helped to increase the flow of participants into clinical trials of the drug. The Foundation also has granted funding to Acadia for studies of a different Parkinson's therapy. 

Acadia is a steering committee member of our Parkinson's Disease Education Consortium. This alliance of biotechnology and pharmaceutical firms supports the Foundation's commitment to furnishing high-quality educational resources for the Parkinson's community. While MJFF's educational offerings are made possible by the generous support of our Parkinson's Disease Education Consortium, content and perspective are solely our own.

The medical information contained in this article is for general information purposes only. The Michael J. Fox Foundation for Parkinson's Research has a policy of refraining from advocating, endorsing or promoting any drug therapy, course of treatment, or specific company or institution. It is crucial that care and treatment decisions related to Parkinson's disease and any other medical condition be made in consultation with a physician or other qualified medical professional.

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