[Tupelo3]

If you have interest in staying on top of the new drug/procedure pipeline for Parkinson's, this report is hot off the press:

America’s biopharmaceutical research companies currently are developing 37 innovative new medicines to help the estimated 10 million people worldwide who are living with Parkinson’s disease. These medicines in development – all either in clinical trials or under review by the Food and Drug Administration (FDA) – include 23 for Parkinson’s disease, 11 for related conditions and 3 diagnostics, according to a new report by the Pharmaceutical Research and Manufacturers of America (PhRMA). -



http://www.phrma.org/sites/default/f...ons-report.pdf

[ashleyk]

I noticed that there are many phase 1 and 2 trials but few phase 3. And application to market a new drug seems to go nowhere with the FDA. Maybe they know what they are doing or maybe they see no urgency to do anything.
In any event there seems to be a drug in phase 3 for advanced PD which seems very helpful for quality of life, safinamide. Is this study going to be put on the shelf or fast tracked by the FDA? People with 10 or more years of PD are running out of time.

http://www.ncbi.nlm.nih.gov/pubmed/24323641
Abstract

Levodopa is effective for the motor symptoms of Parkinson's disease (PD), but is associated with motor fluctuations and dyskinesia. Many patients require add-on therapy to improve motor fluctuations without exacerbating dyskinesia. The objective of this Phase III, multicenter, double-blind, placebo-controlled, parallel-group study was to evaluate the efficacy and safety of safinamide, an α-aminoamide with dopaminergic and nondopaminergic mechanisms, as add-on to l-dopa in the treatment of patients with PD and motor fluctuations. Patients were randomized to oral safinamide 100 mg/day (n = 224), 50 mg/day (n = 223), or placebo (n = 222) for 24 weeks. The primary endpoint was total on time with no or nontroublesome dyskinesia (assessed using the Hauser patient diaries). Secondary endpoints included off time, Unified Parkinson's Disease Rating Scale (UPDRS) Part III (motor) scores, and Clinical Global Impression-Change (CGI-C). At week 24, mean ± SD increases in total on time with no or nontroublesome dyskinesia were 1.36 ± 2.625 hours for safinamide 100 mg/day, 1.37 ± 2.745 hours for safinamide 50 mg/day, and 0.97 ± 2.375 hours for placebo. Least squares means differences in both safinamide groups were significantly higher versus placebo. Improvements in off time, UPDRS Part III, and CGI-C were significantly greater in both safinamide groups versus placebo. There were no significant between-group differences for incidences of treatment-emergent adverse events (TEAEs) or TEAEs leading to discontinuation. The addition of safinamide 50 mg/day or 100 mg/day to l-dopa in patients with PD and motor fluctuations significantly increased total on time with no or nontroublesome dyskinesia, decreased off time, and improved parkinsonism, indicating that safinamide improves motor symptoms and parkinsonism without worsening dyskinesia. © 2013 International Parkinson and Movement Disorder Society.

[Tupelo3]

Originally Posted by ashleyk

I noticed that there are many phase 1 and 2 trials but few phase 3. And application to market a new drug seems to go nowhere with the FDA. Maybe they know what they are doing or maybe they see no urgency to do anything.
In any event there seems to be a drug in phase 3 for advanced PD which seems very helpful for quality of life, safinamide. Is this study going to be put on the shelf or fast tracked by the FDA? People with 10 or more years of PD are running out of time.

.

ashleyk, I don't think Newron Pharmaceuticals, the manufacturer of Safinamide, has any intention of "shelving" the drug. In fact, they have already submitted a Marketing Authorization Application (MAA), to the European Medicines Agency (EMA). Newron is an Italian company so this makes sense. Although I am not aware of their having received a US FDA fast-track designation, the company has stated that they hope to have an NDA filed with the FDA late first quarter or early second quarter of 2014. In their pre-NDA meeting, the FDA did not request any further clinical studies on the drug, which is very positive. Therefore, I would think this drug goes thru the approval process with the MAA and FDA rather "quickly" (which is just a relative term for people who are desperately waiting for something new). The results of the Phase III studies were very positive (significant efficacy and good safety profile), so I think the industry is looking for the drug to be approved by all agencies.

[cbrf1wr]

"The results of the Phase III studies were very positive (significant efficacy and good safety profile), so I think the industry is looking for the drug to be approved by all agencies."

This is ALL that should be needed for immediate approval by the FDA. That is their job. It's obvious that job is not being carried out correctly.

[Jim091866]

Originally Posted by cbrf1wr

"The results of the Phase III studies were very positive (significant efficacy and good safety profile), so I think the industry is looking for the drug to be approved by all agencies."

This is ALL that should be needed for immediate approval by the FDA. That is their job. It's obvious that job is not being carried out correctly.

FDA Frustrate and Delay Agency.