Rumor mill churning re: Vertex drugs

[triples15]

Hi all!

I have recently read a few somewhat concerning things on social media regarding Kalydeco, that I had not previously heard anything about. Instead of spending hours googling trying to glean info, I thought I'd ask y'all (I say y'all now) on here because we have so many super knowledgeable folks. Because I want to get correct info and not perpetuate what is, perhaps, false info, I want to clarify that these things I have read came from peoples' personal facebook posts about it. These aren't from news stories or journal articles etc.

Anyway, the first thing I heard is that many people (men and women) on Kalydeco are having issues with very high testosterone levels? Also, whether related or unrelated, having a lot of hair loss issues? I had never heard of either of these until a couple days ago.

Another thing that people are talking about is a carcinogen study (on Kalydeco I believe) that the results are about to be released soon. It seems almost as if people are expecting a negative outcome there?

The third and final thing I read is that after being told by Vertex they would receive 2 year open-label drug after study participation, people are now being told that is no longer the case and they/their insurance will have to start paying. I'm not 100% sure to which study this was referring, but I believe it was the double-delta study.

Anyway, really just hoping to see if anyone has any solid info on any of these issues.

Autumn 34 w.CF

 

[Aboveallislove]

Hey Autumn,
I hadn't heard any of that and hope it's a matter of one person saying something and with social media it getting shared etc. I'm going to call FDA this morning to see if they've had reports of adverse events along those lines. Not sure if they'll say, but I'd think so. I'd think the hair and testerone would have been reported at the expanded study for Kalydeco (were those for Kalydeco or Orcambi) which was released I think a year ago. I didn't realize they had a cancer study on going, but I'd think with any long term drug they would. On that, I wouldn't think any gossip has a basis in fact because no one would know. Hopefully others have some thoughts and I'll post once I hear from FDA anything.

 

[triples15]

Thanks Love! I just hate reading anything like that because while I'm SO excited about all that's going on, I can't shake the thought that we could still all have the rug swept out from under us at some point. Maybe I am a pessimist at heart?

I did some searching and found the main post I was referring to. I am going to post it with names redacted so that you have more specifics because I don't think I summarized very well and got a couple things wrong:

We were brave enough to do the study and were promised the drug for free for TWO YEARS, well guess what? Vertex called last week to take that back too. Now we have to pay for the drug. The long-term study was cancelled.
So did they rush the drug to market to help everyone? Do we know if the long term effects will merit the cost? It remains to be seen.
The company is suppressing side effect reports too, by the way.
When (name redacted) testosterone levels went from 300 to over 1170, causing severe acne, mood swings and testicular changes, the company denies its the drug doing it. (We aren't the only ones with hormonal changes either, I personally have spoken to three other people who have stopped the drug due to high T. Women and men)
I hope children are being closely monitored for their T levels in the 12 and under trials!

Here is one of the responses to this post:

It's not good that there are side effects that are still not being made public. I know my clinic is not prescribing this until 3 months to see more results as my clinic worries about what is not being said or still unknown.

You know the carcinogen study on Orkambi will be concluded in July. Interested to see what that shows.


And another response:

Even if the Carcinogen study shows nothing, Vertex is now admitting quite a few people have complained of hair loss which is a sign of DHT hormone among others being screwed up. That said I'l still take it because it seems to be a female issue mostly. Men should benefit from increased T up to a point as long as immune system doesn't suffer too much from it.


There are a few more responses, but you get the point. At this point it sounds almost as if some people at this point believe there is some sort of "cover up" going on. I hope for ALL of our sakes that these are isolated incidents and the long-term side effects don't prove to be too much.

Did you find anything out from the FDA?

Thanks again,

Autumn

 

[2005CFmom]

I had heard something about the Carcinogen study was being required, but that is the only part I the rumors that I had heard before. The prescription information about Orkambi does mention that no study was done on Orkambi, but that studies were done on the components. I will copy and paste.

13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility
No studies of carcinogenicity, mutagenicity, or impairment of fertility were conducted with ORKAMBI; however, studies are available for individual components,lumacaftor and ivacaftor, as described below.
Lumacaftor
A 26-week study was conducted in transgenic Tg.rasH2 mice to assess carcinogenic potential of lumacaftor. No evidence of tumorigenicity was observed in Tg.rasH2mice at lumacaftor oral doses up to 1500 and 2000 mg/kg/day in female and male mice, respectively.
Lumacaftor was negative for genotoxicity in the following assays: Ames test for bacterial gene mutation, in vitro chromosomal aberration assay in Chinese hamsterovary cells, and in vivo mouse micronucleus test.
Lumacaftor had no effects on fertility and reproductive performance indices in male and female rats at an oral dose of 1000 mg/kg/day (approximately 3 and 8 times,respectively, the MRHD on a lumacaftor AUC basis).
Ivacaftor
Two-year studies were conducted in mice and rats to assess carcinogenic potential of ivacaftor. No evidence of tumorigenicity was observed in mice and rats ativacaftor oral doses up to 200 mg/kg/day and 50 mg/kg/day, respectively (approximately equivalent to 3 and 10 times the MRHD based on summed AUCs of ivacaftorand its metabolites).
Ivacaftor was negative for genotoxicity in the following assays: Ames test for bacterial gene mutation, in vitro chromosomal aberration assay in Chinese hamster ovarycells, and in vivo mouse micronucleus test.
Ivacaftor impaired fertility and reproductive performance indices in male and female rats at an oral dose of 200 mg/kg/day (approximately 15 times the MRHD basedon summed AUCs of ivacaftor and its metabolites). Increases in prolonged diestrus were observed in females at 200 mg/kg/day. Ivacaftor also increased the number offemales with all nonviable embryos and decreased corpora lutea, implantations, and viable embryos in rats at 200 mg/kg/day (approximately 15 times the MRHD basedon summed AUCs of ivacaftor and its metabolites) when dams were dosed prior to and during early pregnancy. These impairments of fertility and reproductiveperformance in male and female rats at 200 mg/kg/day were attributed to severe toxicity. No effects on male or female fertility and reproductive performance indiceswere observed at an oral dose of ≤100 mg/kg/day (approximately 8 times the MRHD based on summed AUCs of ivacaftor and its metabolites).

 

[Aboveallislove]

Thanks Love! I just hate reading anything like that because while I'm SO excited about all that's going on, I can't shake the thought that we could still all have the rug swept out from under us at some point. Maybe I am a pessimist at heart?

I did some searching and found the main post I was referring to. I am going to post it with names redacted so that you have more specifics because I don't think I summarized very well and got a couple things wrong:

We were brave enough to do the study and were promised the drug for free for TWO YEARS, well guess what? Vertex called last week to take that back too. Now we have to pay for the drug. The long-term study was cancelled.
So did they rush the drug to market to help everyone? Do we know if the long term effects will merit the cost? It remains to be seen.
The company is suppressing side effect reports too, by the way.
When (name redacted) testosterone levels went from 300 to over 1170, causing severe acne, mood swings and testicular changes, the company denies its the drug doing it. (We aren't the only ones with hormonal changes either, I personally have spoken to three other people who have stopped the drug due to high T. Women and men)
I hope children are being closely monitored for their T levels in the 12 and under trials!

Here is one of the responses to this post:

It's not good that there are side effects that are still not being made public. I know my clinic is not prescribing this until 3 months to see more results as my clinic worries about what is not being said or still unknown.

You know the carcinogen study on Orkambi will be concluded in July. Interested to see what that shows.


And another response:

Even if the Carcinogen study shows nothing, Vertex is now admitting quite a few people have complained of hair loss which is a sign of DHT hormone among others being screwed up. That said I'l still take it because it seems to be a female issue mostly. Men should benefit from increased T up to a point as long as immune system doesn't suffer too much from it.


There are a few more responses, but you get the point. At this point it sounds almost as if some people at this point believe there is some sort of "cover up" going on. I hope for ALL of our sakes that these are isolated incidents and the long-term side effects don't prove to be too much.

Did you find anything out from the FDA?

Thanks again,

Autumn

i played telephone roulette for about fifteen minutes...got through only to be put in someone's voicemail...left detailed message and will definitely update when I hear back...or call again if nothing by Monday. Thanks for added details!

 

[Aboveallislove]

just got off the phone with the FDA. They confirmed that all side effects must be reported by the company during the trial...Vertex can't just say it isn't related to the drug. Vertex and the FDA will then explore together to determine if it is related to the drug. And if there were a connection at 12/2014 when the label was updated re cataracts the label would also state the adverse reaction. There is nothing about high T levels or hair loss at that time (or obviously in the trials, that was considered related to the drug by the FDA). If anyone individually has reported an adverse event, that can be found out by making a FOIA (Freedom of Information Act Request) at 1-301-796-3900, which then might take some time. And I have never heard the comment re hair loss from Vertex and that has never been reported as far as I know in any of the sideffects (I asked the FDA that and she said no). So my take on all of this is that it is mostly rumor.