New Hope for People with Sickle Cell Anemia as FDA Approves Drug Written by Moira McCarthy on December

The new drug Oxbryta attacks the underlying causes of sickle cell anemia, not just the symptoms. Getty Images
  • The FDA has granted “fast track” approval for the drug Oxbryta to treat sickle cell anemia.
  • The medication works by preventing abnormally shaped red blood cells from restricting the flow in blood vessels.
  • People in the sickle cell anemia community say they’re thrilled with the approval, because Oxbryta treats the underlying causes of the blood disorder and not just the symptoms.
A new drug that’s the first to treat the underlying causes of sickle cell anemia rather than just the symptoms will hit the market within days.
That’s thanks to a decisionTrusted Source by the Food and Drug Administration (FDA) to grant accelerated approvalTrusted Source for the medication.
Those in the sickle cell anemia community say they’re thrilled that the drug Oxbryta has been quickly approved.
“There is so much excitement about this. It’s almost feverish,” Beverley Francis-GibsonTrusted Source, president and CEO of the Sickle Cell Disease Association of America, told Healthline. “It’s been a long fight and a long haul in the sickle cell community.”
Oxbryta (also known as voxloter) is a product of Global Blood Therapeutics (GBT) of San Francisco.
The FDA’s decision to fast-pace the drug to market came as the result of positive responses to the drug seen in participants in clinical trials. It’s the second drug to treat sickle cell anemia to come to the market in the past month.
In mid-November, the Novartis drug Adakveo was approved by the FDA to reduce the “frequency of sickle cell pain crises.”Ho
Sickle cell anemia, also called sickle cell disease, is a chronic, inherited blood disorder in which red blood cells are abnormally shaped in a crescent, or “sickle,” shape.
Those cells restrict the flow in blood vessels and limit oxygen delivery to the body’s tissues, leading to severe pain and organ damage.
The blood disorder is also characterized by severe and chronic inflammation, which causes episodes of pain and organ damage.
Oxbryta works by preventing red blood cells from slowing down blood flow.
“It is always exciting to be able to provide a new treatment option for patients with a serious and life threatening condition such as sickle cell disease,” Brittney Manchester, an FDA press officer, told Healthline.
In the case of Oxbryta, Manchester says the approval was based on the results of a clinical trial in which 51 percent of the participants who took Oxbryta saw an increase in hemoglobin response rate.
This is significant. Oxbryta is an inhibitor of deoxygenated sickle hemoglobin polymerization, which is the central abnormality in sickle cell anemia.
That approval, Manchester says, was cause for celebration at the FDA.
“When we are able to expedite the review of drugs to treat serious conditions and fill an unmet medical need, we are able to get important new drugs to patients earlier,” she said.

GBT officials announced in late November that the drug should be available via the company’s specialty pharmacy partner networks.
Ted Love, MD, the president and CEO of GBT, told Healthline they’re still on track to make that happen.
“The response from the patient community has been very positive, indeed very emotional,” he said. “There is a very real sense that the long-awaited approval of new therapies is a true turning point for a community that has struggled with a lack of innovation and limited access to care for decades. The feedback we’ve received is that there is strong interest from the patient community to talk to their physicians about the potential to try Oxbryta.”
Physicians are also jubilant about the news.
“The news of the FDA approval of Oxbryta is causing me immense excitement,” Maureen Achebe, MD, director of Brigham and Women’s Sickle Cell Disease Clinic in Brookline, Massachusetts, told Healthline.
Achebe says that while providers have a good grasp on the “many moving parts of managing the disease,” frustration has come with the lack of options for treatment.
“The cause of the frustrations providers and patients with [sickle cell anemia] may feel stem from the lack of specific disease-targeted medications. The result of this is a medical system that only has the ability to treat [sickle cell anemia] patients with symptomatic remedies.
“Now, since the hallmark manifestation of sickle cell disease is excruciating pain, patients are treated with opioid medications. In general, any scenario in which the long-term treatment strategy is anchored on chronic opioid medications is bound to have a suboptimal outcome,” Achebe said.Lo
Achebe also sees this as a trend with more being discovered, understood, and developed for treatment of the blood disorder.
“There has definitely been an uptick in the attention being paid to [sickle cell anemia], both by professional organizations and by pharmaceutical companies,” she said.
Achebe points to the American Society of Hematology (ASH) decision in 2016 to place an emphasis on advancing the care of sickle cell anemia as a major reason.
“ASH’s [sickle cell anemia] initiative addresses the state of [sickle cell anemia] treatment in the U.S. and globally to improve care, early diagnosis, treatment, and research. At the same time, pharmaceutical companies are working on new compounds targeting SCD at an unprecedented speed
“The future of treating sickle cell disease looks bright. We are excited by the potential array of different treatments that will be available to choose from. The more options there are, the more likely that a suitable treatment(s) will be found for each patient,” Achebe said.
“There is a feeling of immense hope,” added Francis-Gibson. “Every time a new drug comes along, it is one more opportunity to cut hospital stays, to alleviate pain, and to extend life.”

The drug will come at a cost.
The treatment will be priced at $10,417 per month, or around $125,000 per year.
GBT has launched a program to help people, including those on Medicare, to get insurance coverage for the drug.
“Ensuring that patients are able to gain access to Oxbryta is our top priority,” Love said. “As part of this comprehensive support program, we have a team of highly trained professionals who truly care about making a difference in sickle cell disease who will provide personal assistance to help patients navigate the access process. We expect Oxbryta to be widely available, and are working with both public and commercial payers to expedite coverage.”
Achebe adds that the people who participated in clinical trials are owed a debt of thanks.
We owe a great deal of gratitude to the [sickle cell anemia] patients who participate in research and clinical trials of compounds for this disease,” she said.
“Our successes may or may not benefit the participants themselves. However, they benefit the larger population of [sickle cell anemia] patients and future generations of [sickle cell anemia] patients.”

Post a Comment