Daratumumab Expanded Access Program Initiated
A new program has been started that will make it easier for relapsed multiple myeloma patients in the United States to be treated with the investigational drug daratumumab.
Johnson & Johnson (NYSE:JNJ), which is developing daratumumab in collaboration with the Danish biotechnology company Genmab, has initiated an “expanded access program” – a special kind of clinical trial – to broaden opportunities for qualifying U.S. myeloma patients to be treated with the drug.
The program is expected to remain in place until a decision is made by the U.S. Food & Drug Administration (FDA) regarding the new drug application Johnson & Johnson is currently submitting for daratumumab. The company announced last month that it had started the new drug application process for daratumumab, and The Beacon expects an FDA decision on the application by March 31, 2016 (see related Beacon news).
Until the FDA reaches a decision on daratumumab's application for approval, the drug will only be available to myeloma patients who participate in clinical trials testing the treatment. The new expanded access program for daratumumab is intended to supplement existing daratumumab trials so that more relapsed myeloma patients have access to the drug prior to the FDA’s decision on the drug’s approval application.
Patients in the expanded access program will be able to be treated with daratumumab for as long as they respond to the drug, or until they experience side effects that preclude further treatment with the drug, or until the program is stopped.
Daratumumab is the only myeloma therapy, however, that patients may receive while they are participating in the program. Combined treatment with other myeloma therapies, including steroids such as dexamethasone (Decadron) or prednisone, is not permitted while a patient is taking part in the program, except for small steroid doses to prevent, for example, possible reactions to the daratumumab infusions.
Eligibility Requirements For The Program
To qualify for the expanded access program, myeloma patients must have experienced disease progression on their current treatment regimen or following their most recent myeloma therapy. For patients with a measurable M-spike, an increase in the M-spike of at least 0.5 g/dL is considered disease progression. For patients without a measurable M-spike, an increase of at least 100 mg/L in the absolute difference between a patient’s kappa and lambda free light chain levels is considered disease progression.
There are also program eligibility requirements related to how many previous treatment regimens a patient has had and what drugs were included in those treatment regimens.
In particular, patients must have been previously treated with at least one drug from the proteasome inhibitor class of therapies – which includes Velcade (bortezomib) and Kyprolis (carfilzomib) – and at least one drug from the immunomodulatory class of therapies – which includes Revlimid (lenalidomide), Pomalyst (pomalidomide, Imnovid), and thalidomide (Thalomid).
Patients also must have had a total of at least three “lines of therapy” to take part in the program. A single “line of therapy” is often equivalent to a single treatment regimen. However, when a patient receives two or more treatment regimens as part of a planned sequence of therapies, the multiple regimens are usually considered a single line of therapy. This is the case, for example, when a patient receives a two- or three-drug combination regimen as initial therapy for their multiple myeloma and then undergoes a stem cell transplant immediately after their initial treatment. The initial therapy and subsequent transplant are considered a single line of therapy.
There is one exception to the requirement that patients have received three prior lines of therapy to be eligible for participation in the expanded access program. Patients who are “refractory” to at least one proteasome inhibitor and at least one immunomodulatory agent are also eligible for the program. A patient is considered to be refractory to a treatment if they do not respond to it, or if their disease progresses within 60 days of stopping the treatment.
This exception would apply, for example, if a patient received a combination of Pomalyst, Velcade, and dexamethasone as their second line of therapy, and either failed to respond to it, or their disease progressed within 60 days of stopping the treatment. In this case, the patient would be eligible for the program, despite having only two prior lines of therapy, because they were refractory to both a proteasome inhibitor (Velcade) and an immunomodulatory agent (Pomalyst).
There are some program participation restrictions related to potential daratumumab side effects. Myeloma patients may not take part in the program, for example, if they have chronic obstructive pulmonary disease (COPD) that has lowered their lung function by more than 50 percent, or if they have had persistent moderate or severe asthma in the last two years.
More About The Program And Daratumumab
The expanded access program is being offered through myeloma treatment centers in 48 different locations throughout the U.S. There will be no cost to patients or their insurance companies for the daratumumab they are treated with during the trial. There are likely to be other charges, however, for which patients and/or their insurance companies will be responsible, such as infusion fees and lab tests.
Daratumumab is a monoclonal antibody that binds to a protein known as CD38, which is commonly found on the surface of myeloma cells. Once bound to a myeloma cell, daratumumab then attacks the cell while also signaling the patient's immune system to act against the cells.
Two other CD38 monoclonal antibodies are also under development as potential myeloma therapies: SAR650984 and MOR202. In addition, elotuzumab, a monoclonal antibody that targets a different protein found on myeloma cells, is also being investigated as a potential myeloma therapy.
Updated data related to daratumumab’s efficacy and safety were presented at this year’s American Society of Clinical Oncology and European Hematology Association annual meetings (see related Beacon news). These results have generally impressed myeloma specialists, due both to the drug’s activity as a myeloma therapy and its safety.
For more details about the daratumumab expanded access program, see the detailed description and related information at the clinicaltrails.gov website.
Questions about an individual patient’s eligibility for the program are best addressed by the patient’s physician or by representatives of Johnson & Johnson, who can be reached using the contact information provided at the clinicaltrials.gov page for the program.
Related Articles:
- None Found
This is fantastic news for many multiple myeloma patients. I am currently on the daratumumab trial (32nd visit) at Dana Farber and am so happy to have been a part of this wonderful trial! I am going strong after almost six years – RVD, SCT, RD, RVD, Pomalyst trial, radiation and now Dara. Feel better than I have in 6 years. Get excited, folks. Trials help us all and they are closely monitored!
Sounds real hopeful. Thank you so much for the update. Definitely a trial to keep in mind.
Hi, I would like to know if patients currently outside of the US can access this trial and/or can participate by travelling to the locations in the US.
Thanks for the feedback regarding the article, everyone.
Maria - The expanded access program is being offered only in the U.S. It's possible that a similar program will be started in other areas of the world once an application for daratumumab's approval is filed there. But that's not certain.
It's possible that residents of other countries may be able to take part in the trial by participating at one of the U.S. locations where it is offered. You will have to contact Johnson & Johnson using the information at the clinicaltrials.gov page mentioned in the article to find out whether or not program participants have to be U.S. residents.
Keep in mind, however, that the program requires weekly infusions of daratumumab for the first two months a patient participates in the program, and an infusion every two weeks for the following four months, and those infusions almost certainly have to be done at the participating U.S. treatment center.
Awesome news for my husband! His doctor had recommended he get into a trial of Daratumumab, but he had not been eligible for any of them as he had failed the other drugs that the Daratumumab were being given in conjunction with. Duratumumab given alone will make him eligible. The timing couldn't be better. Husband contacted his doc today and arrangements are starting.
Deb (or anyone else who knows),
What work-up did they have you go through before the trial (imaging studies, bone marrow biopsy,?). Did your insurance company cover those?
Thanks,
Marianna
Please let me know when someone has it up and running. We are also wondering what the platelet requirement will be.
Thanks.
Merikay
Would it be difficult to extend the expanded access program to daratumumab to the 5 existing Canadian test sites of this wonderful drug? I already underwent 4 different therapies that left my body severely damaged. This single drug therapy may help to fight my cancer where the previous poisoneous therapies stopped working.If I will have to wait for the Canadian approval of daratumubab it may come too late for me.
I followed this drug tests from the first reports of the Danish researchers with an intuition of a sick person finding the right drug for him. Even better are the resent reports that Daratumumab is administered without any other required poisonous drug! That may allow my damaged body to partially recover from some side-effect of my previous therapies (damaged nervs in my legs and hands and also heart.
Hi Jan, I know that Daratumumab is not approved yet in Canada. I did not see any Cdn. locations listed in the 'expanded access' program linked to the clinical trials.gov either. Just a thought...could you and your oncologist contact youR Janssen representative for further information about this? I have found the oncology drug reps whom I have met at patient education events to be really concerned about patients. Maybe there is a way you could get access to that drug. Good luck!
Hi Nancy. Do you, please, remember name of the Janssen representative you met at a patient education event? Since 2013, daratumumab has also been tested at 5 Canadian cancer centres and my hope is that the Janssen company may expand their expanded program on all the previously used test centres. Thanks for your helpful suggestion.
The Janssen Canada person I met was the drug rep! Velcade is the myeloma drug that they sell here. I think that your doctor would know who that was in your area. Also, I went on the company's website and in the Oncology section a patient may contact them. Probably if you did that, and explained your situation thoroughly, you could get a response from them. Hope that helps!
There is a trial in Australia at Melbourne's St Vincen'ts Hospital
showing excellent results as reported in the press yesterday.
Does anyone know where this trial is available in the Northeast?
Yes. The trial in Australia showed a great response in one third of patients. Looks promising.
Hi Jan, any luck accessing daratumumab in Canada? I am in a similar situation as you.
Jan, Sam,
I read your posts and I would be interested in hearing more. I am doing some research in the area and would greatly appreciate to hear more.
That's interesting to know! I just had my second transplant and my numbers are beginning to rise. IGG 1665 and so have my lambda light chains....I am currently on maintenance started off with Kyprolis and Pomalyst....was taken off Pomalyst....and Zometa every three months....I was my PA today...but will see my oncologist tomorrow! I will discuss this trail with him. Probably go to MD Anderson...have to look if they have this trail going on.
I am refractory/relapse MM. I am IGG multiple myeloma, Lamda light chains stage III.
I've had 2 SCT radiation to many parts do my body, Modified CVADS, and most of the drugs to treat MM. I was diagnosed with MGUS 2000. MM 2006 1st SCT 2013, modified CVADS 2014 2nd SCT 2015.
Was interested in immunotherapy too! Have to talk to my oncologist next week...but good to know new treatments are out there for me and my stage of multiple myeloma!
So I want to thank all the research doctors and oncologist specialist that treat multiple myeloma and nurses that care for the patients...and anyone else that helps....the caregivers and the starting nurses everyone!
Thank you from the bottom of my hear,
Frances Smith!
Get new Myeloma Beacon articles by email.