Getting frustrated with the delay approval of ropeg In the US

[markgenious1981]

Sometimes I can't help to think if enough is being done for MPN sufferers. According to multiple research ropeg has shown superiority over hydroxyurea with Ultimate possible significant molecular response in a selective number of MNP patients. So why there's a delay in getting access to this drug? Why are doctors pushing hydroxyurea on patients and when you refuse they become indifferent? The medical and pharmaceutical fraternity seems to have other interests than meeting the needs of diseased population.

[hunter5582]

Ropeg is in the final stages of approval. There was a delay in part due to the COVID crisis interfering with inspection of the plan that will make the Ropeg. There is a last set of questions to be answered as well. Last I heard, approval is expected before the end of the year. Ropeg does have Orphan Drug status and can be accessed in the USA if your insurance will cover it.

I was waiting for Ropeg, but decided to start on Pegasys to deal with some active symptoms from the PV. So far it is going well. I plan to switch to Ropeg when it becomes available. I could not tolerate HU, so it was either Pegasys or Jakafi. Getting the PEG approved was no problem

HU has been around a long time and most docs are more used to prescribing it. It is the cheapest and easiest med to get approved. It is accepted practice and many docs are not as familiar with the newer forms of PEG-IFN.

It is up to patients to educate themselves about the options and advocate for the treatment options they wish to pursue. This is both our right and our responsibility. We need to work with our treatment team collaboratively and form a relationship of mutual respect. Providers who cannot work this way and treat patients with indifference can and should be replaced.

Like you I am hoping for Ropeg to be available soon. Meanwhile I am blessed to have a wonderful treatment team who works with me, is supportive, and respects by right to make decisions.

All the best.

[markgenious1981 in reply to hunter5582]

Thanks for the reply Hunter. I know there's not much I can do than to wait for the approval, hoping that while waiting I don't progress into endstage.

[hunter5582 in reply to markgenious1981]

You do have the option to start on Pegasys and switch to Ropeg when it gets approved. That is what I decided to do, Working well so far.

[lynxfluff in reply to markgenious1981]

I can’t wait until Ropeg is approved… BCBS Texas refuses to cover Pegasys, even though it meets and exceeds their off label use requirements.

If some of your hesitancy about Pegasys as a stopgap until Ropeg is approved is insurance coverage and copays, Genentech has a patient assistance program that can cover Pegasys for people who meet the income requirements. (Which are totally reasonable - if I recall, it’s $75k for an individual or $150k for married filing jointly.)

That’s what I ended up having to do for 2021. They’ll send you the med for free up to three years, which should give you plenty of time for Ropeg to be given the green light. That’s what I’m banking on, lol.

[markgenious1981 in reply to lynxfluff]

Thanks Lynxfluff. This is very useful information to me

[SuET2017 in reply to hunter5582]

Hi, I’m ET triple neg and could not tolerate HU or anagrelide and am now on peg-inf with some side effects but tolerable. What is the benefit of ropeg over peg-inf?

[hunter5582 in reply to SuET2017]

Ropeg only has to be injected every 2 weeks. Due to its slower release, it may be easier to tolerate. It will also be FDA approved for PV, so it may be easier to get approved.

[Buggerbear in reply to hunter5582]

Hunter, I am just curious as to why you chose Pegasys over Jakafi for the PV. I am currently taking the Jakafi for my PV and ET. I am sure you have reasons as you are a very smart and informed member of this very helpful website. Thanks so much!

[hunter5582 in reply to Buggerbear]

Just saw this note from a while back. I chose PEG over Jakafi for a couple of reasons. One of which is that I do not experience pruritis or any of the other symptoms RUX is particularly good for. I also believe that PEG can be disease modifying in a way the other options cannot match. I also have the NF1 mutation that puts me at increased risk of progression to AML. Once it became clear that the phlebotomy only approach was not optimal, PEG was the optimal choice. I did also try to get into the rusfertide clinical trial but did not qualify.

Hope you are doing well. All the best.

[Buggerbear in reply to hunter5582]

Thank you Hunter for your reply Hunter. Hope you continue to do well with the PEG!!

[EPguy in reply to hunter5582]

Rusfurtide I've read has FDA's top "breakthrough" designation and very few side effects. So it should go fast as possible. I understand it's great replacement for phleb, while keeping iron in control. But does it benefit WBC or PLT if these are also high?

[hunter5582 in reply to EPguy]

Rusfertide is a hepcidin mimetic. Hepcidin is part of the body's iron metabolism system that keeps iron locked in storage and not available for erythrocytosis. It controls erythrocytosis while letting the body retain more iron. This obviates the need for phlebotomies and reducing the symptom burden cased by iron deficiency.

Rusfertide would not directly affect thrombopoiesis nor leukopoiesis. It is true that iron deficiency can drive up thrombocytosis. It did for me by about 200K. I am not aware of any data that would indicate it would help with this issue, but it seems reasonable to think that it could. Time will tell as we learn more about Rusfertide.

I agree that Rusfertide is one of the most promising developments in managing PV. It could be a significant improvement in quality of life for people with PV. Many people with PV only need to reduce erythrocytosis (keep HCT < 45%/43%). Achieving this goal with a medication that seems to have so few adverse effects would be a significant benefit for many.

[EPguy in reply to hunter5582]

It seems US doctors are resistant to IFN. Maybe because the early IFNs were not well tolerated. Ropeg looks very promising in the Euro trials, with improved tolerance and with good allele reduction. But it takes a few years to really see the allele reductions. It also gives symptom reduction in my reading, something HU is not very good at.

For those of us who get good blood results from HU it may be harder to convince the Dr and insurance to switch to Ropeg. But I am most eager to do so.

As you said, the FDA has been delayed, they need to get to Taiwan (where it's made) for the inspection so it was rejected in March '21. It's supposed to have its next FDA action by Nov 13 2021 with FDA visiting in Aug or Sept. I have no update lately.

[hunter5582 in reply to EPguy]

I think your read on doc resistance is correct. Many are not familiar with how much better the lower doses of the PEGylated forms of IFN are tolerated.

It is much more expensive than HU. In some formulates you have to fail on the cheaper HU before you can access it. That is unfortunate since it is recognized as the other first line treatment option for ET and PV. I definitely prefer the risk/benefit profile of PEG over HU. For the record, I did try HU and I am intolerant of it.

Sure hope we can access Besremi in the USA soon. It will be a huge step forward for MPN treatment. Hopefully we will see rusfertide soon too.

[markgenious1981]

I have an appointment with my hematologist in few days. I will definitely advocate for myself to have the Pegasys until ropeg is available. Thank you hunter for this peice of information.

[Pte82 in reply to markgenious1981]

Hey mark, just a followup on your earlier post mentioning paresthesia. This link mentions thiamine deficiency as a possible cause. Thiamine requires adequate magnesium to change it to it's active form. Check out these forms of thiamine: Benfotiamine, TTFD and Sulbutiamine as they offer important advantages. They work better with the other B's. There are anti thiamine factors to be aware of that diminish thiamine noted in the second link. . The role active thiamine plays in the brain and body and expecially the vagus nerve must be understood to appreciate the consequences of a deficiency. Consult with your health care provider before using any supplement.

healthline.com/nutrition/th...

stuttersense.blogspot.com/2...

[markgenious1981 in reply to Pte82]

Thank you Pte82. I will definitely mention this to my doctor and do my own investigation as well

[EPguy]

I've been following the news in Taiwan where ropeg is made. Here is a translated story from a Taiwan web site udn.com that confirms potential good news that our needs are on the radar and a rare overseas FDA inspection is happening. I had mentioned above about it, now a real update. (I hope it is not too long to post)

Sept 08 2021

Since the outbreak of the epidemic, the US Food and Drug Administration (FDA) has greatly reduced the number of overseas factory inspections. On the 25th of last month, it reiterated that it would not go abroad unless it is mission critical, which caused delays in the listing of many pharmaceutical companies around the world in the United States. Yaohuayao (6446) has recently broken the convention. Two FDA officials arrived in Taiwan last Saturday (4th) and are expected to conduct factory inspections on Yaohuayao at the end of September. The company aims to obtain US marketing approval for the new drug in November.

FDA officials said last month that from March 2020 to March this year after the outbreak, the FDA conducted a total of 821 mission-critical inspections, of which only 29 were overseas. According to official data, before the outbreak, the FDA conducted annual inspections at home and abroad. The number of factory inspections is more than 1,600.

Huang Zhenggu, general manager of Yaohua Pharmaceutical, confirmed yesterday (7) that after the two FDA officials arrived in Taiwan last Saturday, they are currently under quarantine at an epidemic prevention hotel. Two weeks later, they will go to Yaohua Yao’s Taichung factory to inspect the factory. The entire inspection process is estimated to be one week. Time, the results will be notified on the last day of the factory inspection. If it goes well, Yaohuayao will be able to announce on the same day whether there are no major defects.

Huayao has developed a new type of interferon P1101. In recent years, it has actively applied for marketing authorization in the United States for polycythemia vera. However, in March this year, it received an FDA request for a supplement, suggesting to modify the "Patient Use and Operation Instructions" for the syringe in the imitation list. Test; until June 4, when the FDA notified again, the review can be completed on November 13 this year.

Huang Zhenggu stated that the treatment project PV of the new drug that Huayao applied for listing is a rare disease, which belongs to the unmet medical need (Unmet Medical Need) and meets the FDA's "critical mission" qualification. Therefore, the FDA sent special personnel to Taiwan to inspect the factory.

Huang Zhenggu explained that the Taichung plant of Yaohua Pharmaceutical is a P1101 API manufacturer. In the past, it has passed the EU EMA twice and the Taiwan Food and Drug Administration conducted two factory inspections before listing. In order to apply for the listing of P1101 in the United States, the FDA has completed the inspection of the P1101 filling foundry. The US subsidiary of Yaohuayao has also passed the FDA’s GLP certification. This time FDA officials came to Taiwan to inspect the factory, which can be said to have obtained the US drug certificate. For the last hurdle, Yaohuayao also hired two retired former FDA officials to come to Taiwan to perform the exercise at the Taichung factory.

[Manouche]

I can’t agree more with you Mark. Some would say that ropeginterferon is still experimental, that’s why it’s important to wait. 🙄..Remember what Hasselbalch said in 2014:

« In this context I am worried by the reported claims by Dr. Ayalew Tefferi that IFN is no better than HU and busulphan in the MPNs. If Tefferi has said so, it is problematic that a colleague, who may be aware of his great impact on the “scientific community ” and MPN-patients’ care, stands up and not tells the truth.

Dr. Richard Silver and I and others have during the years delivered a large number of papers – studies and reviews – documenting that interferon is not ” an experimental drug” to be used only in selected cases. Interferon is an old horse in the circus which – for unknown reasons – has been neglected, dismissed and thrown away. »

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