U.S FDA approved new medicine spinraza that contains active ingredient nusinersen for the treatment of rare muscular disorder known as spinal muscular atrophy (SMA). FDA approves this drug in parenteral dosage form and a medical specialist is required to inject this drug because it is administered into the fluid surrounding the spinal cord.
Spinal muscular atrophy is a rare kind of genetic disorder. Motor neurons are important to control motor functions of body like moving, driving, various muscular movements etc. Increase in number of dead motor neurons makes the co-ordination weak between brain and skeletal muscles. This will ultimately leads to the development of SMA (spinal muscular atrophy). However, certain risk factors that play important role in the development of SMA (spinal muscular atrophy) are genetic predisposition, age factor, auto-immune disease etc.
This drug belongs to antisense oligonucleotide family and can be used in all types of spinal muscular atrophy. It is injected through intrathecal route so, much care is required during its administration in order to avoid nerve damage. This drug has been received orphan drug designation which provides incentives to assist and encourage the development of drugs for rare diseases.
Spinraza/nusinersen Side effectsDuring initial clinical trials drug shows following side effects
- Thromobocytopenia (low platelets count)
- Anemia (low RBC's count)
- Increased risk of respiratory tract infection
- Kidney damage (nephrotoxicity)
- Brain damage (neurotoxicity)
- Hypersensitivity reactions
- Skin rashes
- Painful administration
Spinraza/nusinersen Uses or Prescribing informationU.S FDA approved this drug for all kinds of spinal muscular atrophy (SMA). This drug should be used once every 4 months after loading dose administration via intrathecal route. Care should be taken while injecting this drug to avoid any nerve damage.
Spinraza/nusinersen DosageThis drug is manufacturing as single dose vial with potency of 12mg/5mL. Dosage of spinraza is diving into two phases.
Loading Dose: It includes 4 doses each of 12mg intrathecally. Administer first 3 doses at 14-day intervals and then administer 4th dose 30 days after the last (3rd) dose.
Maintainance Dose: It includes dose of 12 mg intrathecally per administration after every 4 months.
This drug is administered by your healthcare practitioner (HCP), which usually means:
- It may comes under special items category.
- You cannot fill this prescription in a regular pharmacy. Your doctor, hospital, or clinic will provide this medication because of possible complications or other issues.
- This drug is likely to be covered under your medical benefit if you have insurance.