Updated: Sep 24, 2020
By Abigail Johnston
Piqray (Alpelisib) was the first new drug application (NDA) for a new molecular entity approved under the Real-Time Oncology Review (RTOR) pilot program on May 24, 2019. This application also used the Assessment Aid (AAid). With these two pilot programs, the approval of Piqray came approximately three months ahead of the PDUFA VI deadline of August 18, 2019. What does that mean? Well, it means that the Food and Drug Administration (FDA) is taking very very seriously the need to fast track medication that looks promising and has both developed and implemented programs to make that happen.
Piqray was approved to be used in combination with fulvestrant for postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer as detected by an FDA-approved test following progression on or after an endocrine-based regimen. The approval of Piqray was based on SOLAR-1 (NCT02437318), a phase 3, randomized, double-blind, placebo-controlled trial of alpelisib plus fulvestrant versus placebo plus fulvestrant in 572 patients including postmenopausal women, and men, with HR-positive, HER2-negative, advanced or metastatic breast cancer whose disease had progressed or on or after receiving an aromatase inhibitor. The study demonstrated that the group receiving Piqray had eleven (11) more months before progression (PFS=progression free survival) versus the group not receiving Piqray and there is no information yet on long term or overall survival (OS).
The most common reported side effects on the alpelisib plus fulvestrant arm were increased glucose, increased creatinine, diarrhea, rash, decreased lymphocyte count, increased gamma glutamyl transferase, nausea, increased alanine aminotransferase, fatigue, decreased hemoglobin, increased lipase, decreased appetite, stomatitis, vomiting, decreased weight, decreased calcium, decreased glucose, prolonged activated partial thromboplastin time (aPTT), and alopecia. The recommended alpelisib dose is 300 mg (two 150 mg film-coated tablets) taken orally, once daily, with food. When given with alpelisib, the recommended dose of fulvestrant is 500 mg administered intramuscularly on days 1, 15, and 29, and once monthly thereafter.
That’s the official information about Piqray.
I’ve been taking Piqray since August of 2019 and I currently am one of the administrators of the Facebook group for people taking Piqray, which includes individuals with other kinds of cancer and people who just have the mutation, PIKC3A, with no cancer. The varying experiences have been fascinating, especially since we have many countries represented in the group as well. What I can tell you from the group is that Piqray has its pros and cons, just like any other cancer drug. For those of us who were prescribed Piqray as the first in our doctor’s offices, we call ourselves #PiqrayPioneers. Most of us had to figure out how to deal with the medication and its side effects largely on our own since most of the doctors weren’t fully equipped to help us and that’s been a gift to the patients newly taking Piqray. I’ve seen how patients help each other and how much we have to offer each other in this and other support groups. If you are a patient taking Piqray, I’ve included a link to the support group below.
Another interesting development is that managing the side effects of Piqray usually includes bringing new people onto the team who aren’t usually as fluent in cancers. Since Piqray often causes hyperglycemia (high sugar levels), most of the people taking Piqray need to see an endocrinologist. Endocrinologists are experts in helping patients manage their blood sugar; however, not usually in the context of cancer. Once we started hearing that the endocrinologists needed more training and information, the patient group turned to Novartis and asked for physician education to be developed for doctors other than just the medical oncologists. This is in progress.
Additionally, since diet changes are necessary (most of us on Piqray stick with a modified keto diet) and weight changes can be significant on Piqray, engaging a nutritionist who is skilled in working with cancer patients has also been a key addition to the team. For me, since both Piqray and the medication I take to help with my blood sugar, Jardiance, causes weight loss, I’ve struggled with maintaining a healthy weight.
Talking with my endocrinologist and my new nutritionist has helped me adjust my caloric intake to address my weight loss.
There are some additional studies looking at other medications in this class, but for now, Piqray is the only FDA approved medication to target the PIKC3A mutation.
Here are some additional sources:
See the Piqray FAQs
About the Author:
In 2017, at the ripe old age of 38, while living in Orlando, Florida, I was diagnosed with Stage IV Metastatic Breast Cancer after feeling a lump in my left breast while tandem nursing my boys, who are now 7 and 5. The summer of 2017 was a rollercoaster of emotions as I faced four body and life-changing surgeries amidst chemo and radiation. Once the whirlwind had settled a bit and I’d completed transitioning my clients and staff at my law firm to another law firm, my husband, two boys and I moved to Miami to live with my family. Looking back, I’m astonished at how much has changed, how much we have overcome as a family, and how everyone has adjusted. While I don’t actively practice law, I use my education and training in a variety of ways for the breast cancer community, advocating for patients and thrivers. I have started my own non-profit, Connect IV Legal Services, where I recruit lawyers to do pro bono work for Stage IV patients. I’m also active in many local and national organizations, volunteering and adding my voice to educate and persuade others to connect with and help my community. You can connect with me by checking out my blog, www.NoHalfMeasures.blog, or following me on Twitter, Instagram, Facebook, LinkedIn, or YouTube! - Abigail Johnston