Freedom of information requests

Diagnosis and Treatment of Spinal Muscular Atrophy

Questions

Question 1. How many patients have a current diagnosis for Spinal Muscular Atrophy (ICD-10 Code G12.0, G12.1, G12.8 and G12.9) at your trust?

Question 2. How many patients have been treated in the last 4 months (September to December 2022) with the following products:

Evrysdi (Risdiplam) - total patients
Spinraza (Nusinersen) - total patients
Zolgensma (Onasemnogene) - total patients
Evrysdi (Risdiplam) - new* patients
Spinraza (Nusinersen) - new* patients
Zolgensma (Onasemnogene) - new* patients
*new patients are defined as patients who were not treated with any of Spinraza
(Nusinersen), Evrysdi (Risdiplam) or Zolgensma (Onasemnogene) in the previous 4-
month period (May to August 2022).

Question 3. Of the total patients treated in the last 4 months (September to December 2022) with Evrysdi (Risdiplam), please provide the number of patients that were treated with Spinraza (Nusinersen) in the previous 4 months (May to August 2022).

Question 4. Of the total patients treated in the last 4 months (September to December 2022) with Zolgensma (Onasemnogene), please provide the number of patients that were treated with Spinraza (Nusinersen) in the previous 4 months (May to August 2022).

Question 5. How many patients have been treated with Zolgensma (Onasemnogene) in the last 12 months (January to December 2022)?

Response

Question 1.

39 in the period Sept 22 to Dec 22.

Question 2.

Pharmacy

Drug	QE	HGS
Evrysdi (Risdiplam) - new* patients	10	0
Evrysdi (Risdiplam) - total patients	10	7
Spinraza (Nusinersen) - new* patients	1	Not held
Spinraza (Nusinersen) - total patients	1	Not held
Zolgensma (Onasemnogene) - new* patients	0	Not held
Zolgensma (Onasemnogene) - total patients	0	Not held

 

Question 3.

Pharmacy

Drug	QE	HGS
Evrysdi (Risdiplam) - new* patients	10	Not held
Spinraza (Nusinersen)	1	Not held

 

Question 4.

Pharmacy

Drug	QE	HGS
Spinraza (Nusinersen)	1	Not held
Zolgensma (Onasemnogene)	0	Not held

 

Question 5.

Pharmacy

Drug	QE	HGS
Zolgensma (Onasemnogene)	0	Not held