Unituxin (dinutuximab) worldwide shortage


(Mark Shirran) #1

Our oncologist has told us there is about to be a worldwide shortage of unituxin - the ch14.18 antibody used in COG protocols. I’m not sure if this has been announced publicly yet and it sounds like information is still scant, but there are grave concerns locally that the shortage may last years! If you are early on in your frontline treatment on a COG protocol, it might be worth checking with your own oncologist about whether the antibody is guaranteed. I’m presuming this shortage doesn’t include the antibody used in SIOPEN protocols/trials (dinutuximab beta). And obviously same for the MSKCC antibody 3F8.


(Donna Ludwinski) #2

Thanks for heads up Mark, this is a HUGE concern and SO important to know for advocates in this arena! I would appreciate any information you might have on this! By the way I was so pleased to meet your sister at the SKC Parent Meeting in London. Let’s connect soon.


(Mark Shirran) #3

Hey Donna,

Yes Emma enjoyed meeting you too!

Unfortunately I don’t have any more details about this. Our oncologist first mentioned it a few weeks ago and he repeated his concerns again this week. He said emails are going back and forth across the world but it looks very likely that there is going to be a shortage for a significant period of time. Given that he first mentioned it a few weeks ago, I had assumed the word would be out by now. Hopefully it’s a false alarm but he did seem very sure about it.

Sorry I can’t be of more help!

Merry Christmas and a Happy New Year,

Mark


(Sam Saffron) #4

This is really scary, we really got to contact the manufacturer to get an official statement.

Stock for united therapeutics is looking fine, so I guess the company is in no kind of financial trouble.

I can not easily find an email for them, perhaps someone US based can try calling http://www.unither.com/contact.html and ask them to respond here? @ludwinski ? @KyleMatthews ?


(Mark Shirran) #5

Yes, I’m surprised that nothing has been made public yet - unless our oncologist is wrong??


(Sam Saffron) #6

The thing I find surprising is that its an FDA approved drug, they charge an enormous amount of cash for it. For example our immunotherapy cost out-of-pocket would have been around $200K US.

Recently they started looking at introducing it into frontline chemo (COG are about to start a trial adding it to round 3 and onwards for frontline chemo)

It seems quite shocking from a purely financial point of view. They are making big money out of this drug.


(Donna Ludwinski) #7

Hi Sam and Mark,

Interestingly, @birdni and I got a rather curt request a week ago for a call with the guy in charge of global distribution – Jay Watson. We talked to him last Feb (along with others at UTC) about getting antibody for children in UK in relapse setting – with IT. They never followed through. We have no idea why he wants a call with us – now set up for 11th of Jan.


(Nick) #8

Hi Mark,

This really would be news. We’ve had a reasonable amount of contact with UTC throughout the UK NICE appraisal process, as Donna says the most recent being a request to speak early in the new year (which was almost like a summons). Nothing like this has ever been mentioned. As we understand there is a European distributor that will be responsible for supply in Europe, and it’s expected to be approved in most medically advanced nations, except England, Wales and Northern Ireland.

There have clearly been goings on at the company. And in October they pulled the plug on a sponsored meeting at SIOP in Dublin with no word of explanation, which raised a few eyebrows.

As part of the deal they did to purchase the commercial rights to dinutuximab they have an ongoing obligation to provide the NCI with Unituxin for its studies free of charge.

I’m more than happy to email them and ask directly, if you want?

Nick


(Donna Ludwinski) #9

@sam @markshirran @birdni – yes Nick – email them!

Mark and Sam – this is who we talked to last Feb

Jay Watson, Executive VP, Strategic Operations
Gil Golden, Head of Medical Affairs
Erny Claxton, AVP, Global Reimbursement & Managed Markets
Marc Dalby (UK Office)

Spoke with Gavin Lindberg and Donna March 1st about chemo + antibody for pts in UK and want help with public perception of UTC in UK; they know that Nick was Expert Consultant for NICE in support of Unituxin:

Gil Golden, MD, PhD
Sr. Vice President and Chief Medical Officer North America
United Therapeutics
ggolden@unither.com

Jay A. Watson, Pharm.D.
EVP Strategic Operations & Logistics
United Therapeutics Corporation
55 T.W. Alexander Drive
P.O. Box 14186
Research Triangle Park, NC 27709
Office: 919.425.8117
Mobile: 919.619.5265
Fax: 919.485-8352
Email: jwatson@unither.com

Erny Claxton
AVP, Global Reimbursement & Managed Markets
United Therapeutics Corporation
55 T.W. Alexander Drive
Research Triangle Park, NC 27709
Cell 407.694.6939
Tel 919.485.8350
Eclaxton@unither.com

Mary and Jeffs came to SKC gala in 2015 (Jeffs was honored) and in 2016 they both left the company. Mary singlehandedly was responsible for the antibody project from beginning of acquiring the license to FDA approval – she was demoted then left.

Mary Smith, PhD
Vice President Product Development at United Therapeutics
MSmith@unither.com

Roger Jeffs
President & Co-CEO at United Therapeutics
United Therapeutics Corporation
55 T.W. Alexander Drive
Research Triangle Park, NC 27709
rjeffs@unither.com

Interesting: Roger Jeffs became co-CEO in January 2015 as Rothblatt agreed to cut her own compensation— will become a senior adviser to the company. He will not stand for re-election to the board when his term expires in June.

“I want to thank Dr. Jeffs and Dr. Zaccardelli for their many contributions to United Therapeutics over the years, and am particularly pleased that Dr. Jeffs will remain involved in guiding the company,” Rothblatt said in a statement.

During the year he was in the position, United reported net income of $652 million, up 92 percent from $340 million in 2014. Revenue reached nearly $1.5 billion, up 14 percent from nearly $1.3 billion in 2014.

The company, which was started 20 years ago as Rothblatt sought pharmaceutical options for her daughter who suffered from pulmonary arterial hypertension, is best known for drugs in that category, including Remodulin, Tyvaso and Orenitram.


(Mark Shirran) #10

I’ve just had it confirmed by an American paeds oncologist (who is a friend of a friend) that they are also expecting the shortage to hit the States early in the New Year. :frowning:


(Kyle Matthews) #11

Can you share who confirmed this to you?


(Mark Shirran) #12

Kyle - the oncologist is at CHOA. I would prefer not to name them on a public forum without their permission.

HNY and well done on achieving the 100k target - amazing effort!!


(Kyle Matthews) #13

Thanks re: the end of year goal! I was pleasantly surprised with our supporters. Incredible.


(Nick) #14

Hi All,

We spoke with UTC yesterday. There is indeed a situation that has arisen where demand has exceeded supply, and the company are facing some tough decisions as a result. They clearly didn’t see it coming, but @ludwinski almost certainly hit the nail on the head when she said it was because of all the kids – many older and therefore requiring larger volumes of ch14.18 – getting a dozen or more rounds of antibody off-study in combination with Temozolomide and Irinotecan in relapse/refractory setting.

In short:
(1) UTC are confident they can meet all ongoing demand in America and Canada.
(2) They have withdrawn from Australia/New Zealand and there is now no route to get antibody there.
(3) They are in dialogue with regulators and official bodies in Europe so the precise situation isn’t yet fully known. However, beyond May/June when the current batch of Unitixun expires there will no longer be EU labelled antibody supply available.

They are not withdrawing from paediatric cancer. They are adding additional bioreactor(s) to their production facility that will quadruple capacity, but manufacturing biologics is a complex process, and it will be between up to 3 years before they are up and running at the new capacity.

  • Nick

(Mark Shirran) #15

Thanks for updating us @birdni.

Mark


#16

Hi - thanks for the update. Two questions/thoughts

1 - 3 years is about average to design, build, commission, and validate a new equipment set - while United build capacity by adding new bioreactors - will they continue to utilise the existing capacity or are they planning on stopping production totally? It’s odd that a company would not build stock using existing capacity befor decommissioning to add new capacity (If indeed they are decommissioning the existing capacity) something is very unusual about that.

2 - Dinituximab beta (ch14.18/CHO) is produced by aperion (isqette was recently acquired by eusapharma) does anyone know if apron are still making beta independent of this supply issue with United?

Alan


(Mark Shirran) #17

As per this thread, it looks like Australasian kids are not going to get the antibody for the foreseeable future (unless they’ve already started their immunotherapy like my son has):

It’s grossly unfair that Australasian kids will miss out whilst supplies have apparently been guaranteed for North American children. By my estimates there will be about 5-15 kids in Australasia that will have been told by now about the antibody shortage (for upfront treatment, not including relapsed patients). It’s not a big number and it would be easy for their voices not to be heard.

I’m therefore interested in starting a media/social media campaign about this. If you are in this unfortunate position and have been told by your oncologist that you/your child won’t be getting the antibody because of this shortage, please make contact with me - either on here or email mark@chasingcharliescure.org

Strength in numbers - let’s get the message out there…


#18

The impacted patients should report this shortage to the FDA. It still appears that the company have not. It would mean they would have to explain what has happened and what their plan is.


(Nick) #19

@pearsona, as we understood it from the call UTC are continuing to manufacture at their existing facility (or facilities). The new bioreactors will provide additional capacity. Nothing is being closed down, it is more that at current full capacity demand is outstripping supply. They also said antibody only has an 18-month shelf-life so there wasn’t stockpiles of it available.

Regarding EUSA, yes their are continuing to make dinutuximab-beta, and take it through the EMA (and NICE) approval processes.

APEIRON no longer play any active role in anything since they sold ch14.18/CHO to EUSA.


(Mark Shirran) #20

Good idea Alan - but do you know if the FDA are interested in hearing about shortages outside of their jurisdiction? I don’t think I’ve seen any Americans post on any of the FB forums that have been affected by the shortage to date? And from what UTC have told Nick and SKC, they are confident of meeting all demand in North America now.