AveXis, a Novartis company, has received approval from the US Food and Drug Administration (FDA) for Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy for pediatric patients with spinal muscular atrophy (SMA), a rare neuromuscular disorder that results in the loss of motor neurons and progressive muscle wasting.

Novartis reports a wholesale acquisition cost of $2.13 million for the gene therapy. The company says that price represents 50% of the 10-year cost of current chronic SMA treatment, which is estimated at $4.1 million, and is 50% below 10-year treatment costs for genetic pediatric ultra-rare diseases, which are estimated at $4.4 million to $5.7 million.

AveXis says it is working closely with payers to offer pay-over-time options up to five years and outcomes-based agreements up to five years as well as providing a patient program to support affordability and access. AveXis has partnered with Accredo to offer a pay-over-time option of up to five years to help ease possible short-term budget constraints, especially for states, small payers, and self-insured employers. In addition, the company says that CuraScript SD has been selected as the sole specialty distributor given its rare disease experience, including gene and cell therapies.

Zolgensma is designed for the treatment of children less than two years of age with SMA. The product is an adeno-associated virus vector-based gene therapy that targets the cause of SMA. The vector delivers a fully functional copy of the human survival motor neuron (SMN) protein gene into the target motor neuron cells, which is a protein critical for the maintenance and function of specialized nerve cells, called motor neurons, according to information from the FDA. A one-time intravenous administration of Zolgensma results in expression of the SMN protein in a child’s motor neurons, which improves muscle movement and function, and survival of a child with SMA.

In another news, Novartis has received FDA approval for Piqray (alpelisib), a kinase inhibitor, for treating certain forms of advanced breast cancer.  Specifically, the drug was approved to be used in combination with the FDA-approved endocrine therapy, fulvestrant, to treat 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 FDA also approved the companion diagnostic test, therascreen PIK3CA RGQ PCR Kit, to detect the PIK3CA mutation in a tissue and/or a liquid biopsy.

Source: Novartis (Piqray), Novartis (Zolgensma approval), Novartis (Zolgensma pricing), FDA (Zolgensma approval), and FDA (Piqray approval)