The Gift of Nucala

2018 is here and as many of you have told me I seemed to have missed one of the most important announcements about the treatment of EGPA/CSS .  I didn’t miss it, I knew everyone else was reporting it so you were all seeing it.  I was waiting for the holiday dust to settle before giving you my take on Nucala.

EGPA patients received a special gift in December

I am very much pro Nucala.   I was on it for 4 months and it stopped my asthmatic cough (which I had for years).  Unfortunately I was one of the few that has a reaction to it and I had to stop using the medicine due to unrelenting joint pain (where I never had joint pain before).  Even though I can not tolerate this medication I think it is a great option for EGPA/CSS patients and have heard many great outcomes from other patients.

On December 12th GlaxoSmithKline  announced that the US Food and Drug Administration (FDA) had approved Nucala (mepolizumab) as the first targeted treatment for Eosinophilic Granulomatosis with Polyangiitis (EGPA), also known as Churg-Strauss Syndrome (CSS).  The new approved dosage is 300mg.  Many patients previously had been approved for the 100mg dose for Eosinophilic Asthma and were using it with success.

GlaxoSmithKline has an excellent patient pamphlet about Nucala on it’s website .  If you are considering Nucala, I would take the time to read it.  Full (more technical) prescribing information can also be found on their site.

What does this really mean to those of us with EGPA/CSS?  What does this replace or used in conjunction with?  It is not a cure but will it improve our quality of life?  There are so many questions about this new drug therapy.

GlaxoSmithKline granted me an interview with their Rheumatologist, Dr. Jennifer Sloane and I was able to ask many questions that EGPA/CSS patients have about Nucala.   I gathered commonly asked questions from Social Media and tried to get answers to Your Questions.

QUESTION: What were the endpoints you were looking for in this study?

ANSWER: Main endpoint was remission was having Birmingham Vasculitis Activity Score (BVAC)  as 0 (zero)  and prednisone less than 4mg.  More patients obtained a Prednisone dose  less than 7 ½ mg but that was not an endpoint.

QUESTION: Will Nucala help with the EGPA even if I do not have asthma?

ANSWER:  All patients in trial had an asthma component to their EGPA/CSS.  GlaxoSmithKline did not study Nucala in patients with EGPA/CSS without asthma. BUT….   There were some other findings in the study that were noted although they were not part of the original endpoints they were looking for.

Sinus?  Where sino nasal relapses – SNOT 22 – Vascultis / Asthma/ Sino Nasal relapse   — Nucala numerically had made it less. Had less relapses in sino nasal category – so Nucala seems to be showing positive results here.

QUESTION: Does Nucala work on blood EOS levels and will it also lower the EOS in organs and in the skin?

ANSWER: In the MIRRA trial, Nucala showed significant reduction in blood eosinophils as early as 4 weeks after the first dose. Eosinophil levels in organ and skin were not evaluated in the MIRRA trial.  It is too invasive to test organs and skin for Eosinophils (EOS).

QUESTION: How will this new dose be administered?

ANSWER: The approved dose is 300 mg subcutaneous injection administered every four weeks (using three separate 100 mg vials) 1-mL injections (at least 2 inches [5 cm] apart if more than 1 injection is administered at the same site.

QUESTION: For the 100mg dose for severe asthma , patients had to go through their pulmonary or asthma specialists.  Which medical specialty will be writing prescriptions for this new 300mg dose?

ANSWER:  Rheumatologists will be able to prescribe NUCALA at 300 mg as will all other healthcare professionals who treat EGPA patients.

QUESTION: Will be there be a financial assistance program for the 300mg dose of Nucala?  If so, where do patients go to find this information?

ANSWER: GSK recognizes that consumers are increasingly being asked by their insurers to take on more cost sharing, making affordability a concern for some patients. Patients seeking help paying for Nucala may be eligible for a copay assistance program offered through Gateway to NUCALA. Patients without insurance may be eligible to receive free medicine from GSK’s patient assistance program. For more information about both programs, patients should contact Gateway to NUCALA at 1-844-4-NUCALA or 1-844-468-2252 (Mon-Fri, from 8 AM to 8 PM ET).

QUESTION: Is there a handout I can print and give to my doctor about the use of the 300mg EGPA dose of Nucala.  (Many doctors may not know about this new dosing option)

ANSWER: Our prescribing information is updated on NUCALA.com, to which healthcare professionals can refer. More educational materials will be available in early 2018. In the meantime, if healthcare professionals require additional information they can contact the GSK Response Center at 1.888.825.5249.

QUESTION: Will Nucala allow patients to totally go off prednisone?  Is it a replacement for prednisone since many patients take prednisone to lower their EOS and lower inflammation.

ANSWER: Patients taking NUCALA in the MIRRA trial were able to significantly reduce their average daily corticosteroid dose compared with placebo as assessed in the last four weeks of the trial. During this time, eighteen percent of patients in the NUCALA group were able to taper off corticosteroids completely versus three percent in the placebo group. All medication adjustments in the MIRRA trial were done with the guidance of the study site investigator.

**NUCALA is not a replacement for prednisone.**

QUESTION: Is Nucala a replacement for Rituxan?  Can Rituxan and Nucala be used safely together?   What can Nucala help me with that Rituxan can’t?

ANSWER: Patients taking Rituximab were not eligible for the MIRRA trial and therefore we didn’t evaluate NUCALA in combination with Rituximab.  Rituximab is not currently approved for EGPA.  It is important to talk with your doctor before switching any of your medicines.

QUESTION:  What are the side effects?  How common are they at the 300 mg level?  Are these side effects more common at 300 mg level?  Are there different side effects at the 300 mg level?

ANSWER: The percentage of EGPA patients experiencing adverse events while taking mepolizumab was comparable between the two treatment groups (97% mepolizumab versus 94% placebo). Injection site reactions (e.g., pain, erythema, swelling) occurred at a rate of 15% in patients receiving mepolizumab compared with 13% in patients receiving placebo. Eighteen percent of patients receiving mepolizumab reported serious adverse events compared with 26% in the placebo group, with the most frequently reported being asthma worsening/exacerbation (3% versus 6%).

As you may be aware, mepolizumab is currently also approved for severe asthma with an eosinophilic phenotype. No additional adverse reactions were identified in the EGPA trial compared to those reported in the severe asthma trials.

QUESTION: Will I have to be on this medication forever? Is there a taper schedule?  Once I stop this medication, How quickly will my high EOS return?

ANSWER: NUCALA was studied in the MIRRA trial for 52 weeks with an 8 week follow up period. We do not have efficacy or safety data about use beyond this duration. During the MIRRA study, patients were not able to taper off NUCALA (unless for safety reasons) so we cannot comment on tapering NUCALA.   Patients should discuss all medication adjustments with their healthcare providers.

QUESTION: How long does Nucala take to become effective?   How quickly does it lower my EOS compared to the quick action of Prednisone?

ANSWER: In the MIRRA trial, there was 83% reduction of eosinophils within four weeks of treatment compared with placebo. We did not directly compare the onset of action of NUCALA with Prednisone.

QUESTION: Does Nucala interact with any other medications? Fertility?

ANSWER: Formal drug interaction trials have not been performed with NUCALA. The impact of NUCALA on human fertility has not been assessed. The average age of a patient diagnosed with EGPA is 48.

I hope these links and information helps you make an informed decision with your healthcare provider if Nucala is right for you.  I am thrilled that we now have in our arsenal of medications that keep EGPA/CSS at bay.    Feel free to share your Nucala stories here and persisting questions.

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