|
|
Post by mnholdem on Sept 23, 2015 7:00:17 GMT -5
17 August 2015
EMA/PDCO/52553/2015
Human Medicines Research and Development Support
EMA Inventory of paediatric therapeutic needs – Endocrinology
Objective of the list
Based on Article 43 of the European Union Paediatric Regulation the Paediatric Committee at the European Medicines Agency (PDCO) is working to establish an inventory to identify the needs in the different therapeutic areas where there should be research and development of medicinal products for children. The inventory is based on the results of a survey of all paediatric uses of medicines in Europe and on the existing list of paediatric needs established by the former Paediatric Working Party; it will be published progressively by therapeutic area. Further information can be found on the EMA website.
Insulines
Product: Inhaled human insulin
Needs:
Data on PK, PD, efficacy and safety in children and adolescents < 18 years.
Data on long-term safety
www.ema.europa.eu/docs/en_GB/document_library/Other/2015/08/WC500192059.pdf
-----
How much longer will Europe need to wait for Afrezza to fulfill this unmet need?
|
|
|
|
Post by peppy on Sept 23, 2015 7:11:05 GMT -5
|
|
|
|
Post by ezrasfund on Sept 23, 2015 7:56:49 GMT -5
The pediatric study is considered Phase 4. Afrezza is already approved. This is just to expand the label to include children 4 to 18 years old.
|
|
|
|
Post by mnholdem on Sept 23, 2015 8:24:13 GMT -5
I believe the data from the FDA pediatric study can also be submitted to the EMA. The first part of the pediatric studies is this Phase I trial which has a target completion date in 2017, then must be followed by the second part, a 52-week Phase 2/3 trial that measures different primary and secondary endpoints.
It's reasonable to expect Afrezza will be approved some time in 2019 for use with 4-17 year old diabetics. It's a shame it must take so long with a drug that has been around as long as human insulin. I also wonder if European pediatric trials would not be as lengthy as in the U.S. Regardless, I'm sure Sanofi will take the path of least resistance.
|
|
|
|
Post by suebeeee1 on Sept 23, 2015 8:26:02 GMT -5
Pediatric use of Afrezza seems like such a no brainer! I understand worrying about what it may do, long term, to lungs. But Geez! I didn't have children with diabetes, but just the idea of needing to stick a young child multiple times a day with a needle, makes me cringe! Kids deserve more.
|
|
|
|
Post by mnholdem on Sept 23, 2015 8:36:42 GMT -5
When I was a kid, at the annual Christmas extended family gathering, the rooms were filled with smoke from cigarettes. Based on what the medical and scientific communities tell us today, we (my brothers, cousins and I) should all be dead. While I agree that drug safety is paramount, I question why it must take so long and involve so many hurdles.
In spite of repeated claims by the FDA that they try to take a higher path, over the past decade there simply seems to be so much evidence to the contrary - evidence that indicates that the REAL drug approval process in the United States is corrupt, overly expensive and easily influenced by deep-pocketed special interest groups.
I forget who they acquired it from, but Sanofi does possess a special FDA Voucher that can be used to designate a drug to be expedited through the approval process. I'd luv to see Sanofi use it for Afrezza's pediatric approval, but that's probably just wishful thinking on my part.
Oh, and my point? The lung is perhaps the most resilient organ in the human body.
----- Perhaps I should pull a Shkreli and send Hillary Clinton an appeal that my poor, suffering daughter may lose her eyesight because of the delays caused by hedge fund managers like Martin Shkreli and their influence on what is increasingly being seen as a corrupt FDA organization.
"Please, Hillary. Would you kiss my baby? Oh, and don't mind that her leg will soon be amputated..."
If Hillary's planning to besiege the drug industry, let's see what she would be willing to do about the inept government agencies whose revolving doors have allowed things to get this far.
------
Incidentally, First Lady Hillary Clinton was heavily involved in children's diabetes research during the two terms that her husband served as President of the United States.
|
|
|
|
Post by brentie on Sept 23, 2015 8:51:48 GMT -5
|
|
|
|
Post by ezrasfund on Sept 23, 2015 11:04:14 GMT -5
www.fda.gov/Drugs/ResourcesForYou/Consumers/ucm143531.htm"Done at hospitals and research centers around the country, clinical trials are conducted in phases. Phase 1 trials try to determine dosing, document how a drug is metabolized and excreted, and identify acute side effects. Usually, a small number of healthy volunteers (between 20 and 80) are used in Phase 1 trials. Phase 2 trials include more participants (about 100-300) who have the disease or condition that the product potentially could treat. In Phase 2 trials, researchers seek to gather further safety data and preliminary evidence of the drug's beneficial effects (efficacy), and they develop and refine research methods for future trials with this drug. If the Phase 2 trials indicate that the drug may be effective--and the risks are considered acceptable, given the observed efficacy and the severity of the disease--the drug moves to Phase 3. In Phase 3 trials, the drug is studied in a larger number of people with the disease (approximately 1,000-3,000). This phase further tests the product's effectiveness, monitors side effects and, in some cases, compares the product's effects to a standard treatment, if one is already available. As more and more participants are tested over longer periods of time, the less common side effects are more likely to be revealed. Sometimes, Phase 4 trials are conducted after a product is already approved and on the market to find out more about the treatment's long-term risks, benefits, and optimal use, or to test the product in different populations of people, such as children."
|
|
|
|
Post by harrys on Sept 23, 2015 11:11:40 GMT -5
I believe the data from the FDA pediatric study can also be submitted to the EMA. The first part of the pediatric studies is this Phase I trial which has a target completion date in 2017, then must be followed by the second part, a 52-week Phase 2/3 trial that measures different primary and secondary endpoints. It's reasonable to expect Afrezza will be approved some time in 2019 for use with 4-17 year old diabetics. It's a shame it must take so long with a drug that has been around as long as human insulin. I also wonder if European pediatric trials would not be as lengthy as in the U.S. Regardless, I'm sure Sanofi will take the path of least resistance. So we are talking about 2019 at the earliest for pediatric approval in the EU? How was this not considered by MNKD and Sanofi earlier... or is that right on track as far as they are concerned? I truly do not understand these timelines, there seems to be no sense of urgency despite MNKDs current position. |
|
|
|
Post by brentie on Sept 23, 2015 11:25:17 GMT -5
I had already seen that but the fact remains that they list it as a Phase I and it has only 46 people in the study. That's not many if you're trying to prove safety and change the label. I hope you're right but I'm skeptical. We'll just have to agree to disagree on this one.
By the way, all the trials they are working on right now are listed as Phase I and were required by the FDA as part of the approval process.
|
|
|
|
Post by mnholdem on Sept 23, 2015 12:36:06 GMT -5
I believe the data from the FDA pediatric study can also be submitted to the EMA. The first part of the pediatric studies is this Phase I trial which has a target completion date in 2017, then must be followed by the second part, a 52-week Phase 2/3 trial that measures different primary and secondary endpoints. It's reasonable to expect Afrezza will be approved some time in 2019 for use with 4-17 year old diabetics. It's a shame it must take so long with a drug that has been around as long as human insulin. I also wonder if European pediatric trials would not be as lengthy as in the U.S. Regardless, I'm sure Sanofi will take the path of least resistance. So we are talking about 2019 at the earliest for pediatric approval in the EU? How was this not considered by MNKD and Sanofi earlier... or is that right on track as far as they are concerned? I truly do not understand these timelines, there seems to be no sense of urgency despite MNKDs current position. FDA Post-Marketing Requirement #1 Required Under: Pediatric Research Equity Act Original Projected Completion Date: 01/31/2021
"An open-label pharmacokinetic (PK), and multiple-dose safety and tolerability dose-titration trial of Afrezza in pediatric patients ages 4 to 17 years (inclusive) with type 1 diabetes (Part 1), followed by a prospective, multicenter, open-label, randomized, controlled trial comparing the efficacy and safety of prandial Afrezza to prandial subcutaneous insulin aspart used in combination with subcutaneous basal insulin in pediatric patients 4 to 17 years old (inclusive) with type 1 or type 2 diabetes (Part 2). Part 2 of the trial should include a 4-week run-in phase and a 52-week randomized intervention phase."
----
After negotiating with the FDA, Sanofi has managed to accelerate the completion dates all three post-marketing trials that are currently active. There is no word on the long-term trial for lung function, originally planned as a five-year study. I think Hakan mentioned at the last cc that Sanofi/MannKind were still working with the FDA on that one.
---- I'm not sure that anyone but Sanofi could tell you if they are planning a separate pediatric trial for Europe. The answer may depend on whether the EU approval track would take less time, but trials still cost $millions so if Sanofi decides to run two pediatric trials simultaneously, that would indicate to me that they expect Afrezza to easily provide payback on their development costs.
|
|
|
|
Post by BlueCat on Sept 23, 2015 15:42:15 GMT -5
When I was a kid, at the annual Christmas extended family gathering, the rooms were filled with smoke from cigarettes. Based on what the medical and scientific communities tell us today, we (my brothers, cousins and I) should all be dead. While I agree that drug safety is paramount, I question why it must take so long and involve so many hurdles.
In spite of repeated claims by the FDA that they try to take a higher path, over the past decade there simply seems to be so much evidence to the contrary - evidence that indicates that the REAL drug approval process in the United States is corrupt, overly expensive and easily influenced by deep-pocketed special interest groups.
I forget who they acquired it from, but Sanofi does possess a special FDA Voucher that can be used to designate a drug to be expedited through the approval process. I'd luv to see Sanofi use it for Afrezza's pediatric approval, but that's probably just wishful thinking on my part.
Oh, and my point? The lung is perhaps the most resilient organ in the human body.
----- Perhaps I should pull a Shkreli and send Hillary Clinton an appeal that my poor, suffering daughter may lose her eyesight because of the delays caused by hedge fund managers like Martin Shkreli and their influence on what is increasingly being seen as a corrupt FDA organization.
"Please, Hillary. Would you kiss my baby? Oh, and don't mind that her leg will soon be amputated..."
If Hillary's planning to besiege the drug industry, let's see what she would be willing to do about the inept government agencies whose revolving doors have allowed things to get this far.
------
Incidentally, First Lady Hillary Clinton was heavily involved in children's diabetes research during the two terms that her husband served as President of the United States.
As someone who also grew up with those once trendy chain-smokers, and is now dependent on bleeding edge pulmonary drugs to stay alive - I don't think the reality is a one-size fits all. That said - with pediatric T1 - the benefits of Afrezza may outweigh the unknown risks (e.g. is this child susceptible to pulmonary disease, and might Afrezza/TS possibly trigger or augment that?) The best case scenario is that pulmonary conditions are also better understood - one of the other top BP markets, and no surprise MNKD is targeting it. That said, I really do hope more focus is spent on cures than on making a buck off of sick people. (or to don the foil hat again, hoping the side effects cause additional sickness to expand additional medically 'adjacent' markets …) |
|
|
|
Post by BlueCat on Sept 23, 2015 15:47:36 GMT -5
Pediatric use of Afrezza seems like such a no brainer! I understand worrying about what it may do, long term, to lungs. But Geez! I didn't have children with diabetes, but just the idea of needing to stick a young child multiple times a day with a needle, makes me cringe! Kids deserve more. LOVE the foil hat! Agreed. I have a kid and even getting the usual pin cushion expected for kids these days is a nightmare. That said, pulmonary disease can easily be as dangerous as diabetes - including in children. I think best case here is that each doctor use careful, good judgment at this point, and that they do these studies right. Reality (I think) is that I don't think from an investor standpoint it'll do a lot for us with stock price - other than help the main revenue target - adult TII - with perception of safety. But I do really hope this comes through for the kids and their families. This would be the best gift from MNKD to mankind to date. |
|
/production/original_573352236.gif)
