New Childhood Cancer Treatment Approved by FDA

David Mitchell, president of Patients For Affordable Drugs, said: “While Novartis’s decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive”.

On Wednesday, the FDA also expanded approval for another drug, tocilizumab, to treat CRS in patients 2 and older.

Novartis’s new gene-modifying cancer therapy’s $475,000-per-patient sticker price has drawn fire from advocate groups calling for cheaper drugs, but analysts said the Swiss drugmaker could initially struggle to break even.

Then, the cells are taken to Novartis’s manufacturing facility in New Jersey, at which point the cells are reengineered to recognize cancer cells and wipe them out.

The immune system has proven a potent weapon for pharmaceutical developers.

“This a brand new way of treating cancer”, said Stephan Grupp, a doctor at the Children’s Hospital of Philadelphia. They are modified to attack other lymphocytes failed, causing the disease, which proliferate quickly.

Novartis said it could have charged as much as $600,000 or $750,000 for the treatment and still been cost-effective.

In view of the success of the clinical trial with 63 patients, the FDA had in 2014 attributed to this gene therapy the status of “advanced therapy” which allows you to speed up the procedures for the placing on the market.

The other big controversy surrounding CAR-T therapy is the significant cost.

Novartis also said it had started working on an “innovative approach” to pricing the drug with the Centers for Medicare and Medicaid Services, the federal agency that administers state-funded healthcare for people on low incomes.

Kymriah, which will be given to patients just once and must be made individually for each patient, will cost $475,000.

Kymriah is approved for use in paediatric and young adult patients with B-cell ALL and is intended for patients whose cancer has not responded to or has returned after initial treatment, which occurs in an estimated 15-20 per cent of patients.

The drug was approved to treat children and young adults up to age 25 suffering from a form of acute lymphoblastic leukemia who do not respond to standard treatment or have suffered relapses.

In July 2017, an FDA advisory panel unanimously recommended approval of the therapy, paving the way for today’s FDA approval. STAT notes that only a few hundred patients per year may be eligible for the cell therapy. The overall remission rate within three months of treatment was 83 per cent.

According to Gottlieb, the FDA has already granted more than 550 active investigational new drug applications related to gene therapy products, including 76 active investigational new drug applications related to CAR-T cell products like Kymriah.

It is not without risks, however, as CAR-T therapies can cause serious side effects such as cytokine release syndrome which can be life-threatening.

The treatment does carry potential side effects, including an immune overreaction, fever, high blood pressure, neurological reactions, acute kidney injury and decreased oxygen.