Drug2nd line

Mycophenolate mofetil

IMPDH inhibitor; suppresses lymphocyte proliferation

Response rate
Effective for maintenance after induction
Onset
Weeks–months
Route
Oral 1-3 g daily
Line
2nd
IgM effect
Partial complement normalization
Evidence level
amber

Evidence summary

Effective maintenance therapy after cyclophosphamide induction. Worm et al. (2000) reported complete resolution of rash, arthralgias, arthritis, and uveitis. However, had no effect on obstructive lung disease. Less toxic than cyclophosphamide for long-term use.

Approved indications

Conditions for which Mycophenolate mofetil has regulatory approval (not specific to rare diseases covered here):

Organ Transplant RejectionLupus Nephritis

Drug identifiers

DrugBankDB00688
ATC CodeL04AA06