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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

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 August 18, 2021: Recently published research in Neurology found that people with Parkinson’s over age 65 who were taking Nuplazid (pimavanserin) had a higher risk of hospitalization or mortality compared to people not taking the drug. The study involved review of medical records, not following people over time. So it’s unclear if the drug causes these risks or if there are other factors playing a role. More people taking the drug were living in long-term care facilities and concurrently taking other medications for psychosis, for example, which could increase these risks and also indicate more significant disease.

Nuplazid’s labeling does include a strong warning about the increased risk of death for elderly people with dementia. As with any medication, it’s important to know about all possible risks and balance with any potential benefits. If you’re taking or consider taking the drug, talk with your doctor.

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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