EUNew treatments and approvals
Oral rilzabrutinib approved in the EU for adults with ITP who did not respond to other treatments
The European Commission approved rilzabrutinib on December 22, 2025.
Official drug approvalITP
Rare Diseases
News
New treatments, research and health policy from around the world and from Türkiye. Each with its source and level of evidence.
45 updates · page 2/3
USANew treatments and approvals
Etuvetidigene autotemcel approved as first gene therapy for Wiskott-Aldrich syndrome
The FDA has approved etuvetidigene autotemcel for Wiskott-Aldrich syndrome.
Official drug approvalWiskott-Aldrich
EUNew treatments and approvals
Nipocalimab approved in the EU for myasthenia gravis, including adolescents aged 12 and over
On November 28, 2025, the European Commission approved nipocalimab for generalized myasthenia gravis.
Official drug approvalMG
USANew treatments and approvals
Intrathecal onasemnogene abeparvovec approved in the USA for SMA patients aged 2 and older
The FDA approved a new form of onasemnogene abeparvovec, given once into the spinal canal (intrathecally), for children, teenagers and adults aged 2 years and older with SMA and a confirmed mutation in the SMN1 gene.
Official drug approvalSMA
USANew treatments and approvals
Plozasiran, which lowers triglycerides, approved in the USA for adults with FCS
The FDA approved plozasiran for adults with FCS, in addition to diet.
Official drug approvalFCS
USANew treatments and approvals
Doxecitine and doxribtimine receive FDA approval as first treatment for TK2 deficiency
The FDA approved the combination of doxecitine and doxribtimine for children and adults with TK2 deficiency whose symptoms began at or before age 12.
Official drug approvalTK2 deficiency
USANew treatments and approvals
Obinutuzumab approved in the USA for kidney inflammation caused by lupus
The FDA approved obinutuzumab as an add-on to standard treatment in adults with active lupus nephritis.
Official drug approvalLupus (SLE)
EUNew treatments and approvals
Oral sebetralstat approved in the EU for hereditary angioedema attacks
On September 17, 2025, the European Commission approved sebetralstat for hereditary angioedema attacks.
Official drug approvalHAE
USANew treatments and approvals
US approval of concizumab expanded to hemophilia A and B without inhibitors
The FDA approved concizumab, previously approved for patients with inhibitors, also for preventing bleeding in patients aged 12 years and older with hemophilia A and B without inhibitors.
Official drug approvalHemophilia
USANew treatments and approvals
Sepiapterin approved in the USA for phenylketonuria; EU approval also in place
The FDA has approved sepiapterin for the treatment of phenylketonuria.
Official drug approvalPKU
EUNew treatments and approvals
EU approves mirdametinib for NF1-related plexiform neurofibromas that cannot be removed by surgery
The European Commission granted conditional approval to mirdametinib for NF1-related plexiform neurofibromas.
Official drug approvalNF1
EUNew treatments and approvals
New once-daily triple CFTR modulator approved in the EU for cystic fibrosis
The European Commission has approved the combination vanzacaftor/tezacaftor/deutivacaftor for cystic fibrosis.
Official drug approvalCystic fibrosis
USANew treatments and approvals
First approval for alkaptonuria in the USA: nitisinone reduces homogentisic acid in urine
The FDA approved nitisinone to reduce homogentisic acid in the urine of adults with alkaptonuria.
Official drug approvalAlkaptonuria
EUNew treatments and approvals
European Commission grants conditional approval to oral givinostat for DMD
The European Commission has granted conditional marketing authorization to givinostat for patients with DMD.
Official drug approvalDMD
JapanNew treatments and approvals
Japan grants conditional approval to DMD gene therapy delandistrogene moxeparvovec
Japan's Ministry of Health has conditionally approved delandistrogene moxeparvovec, for a limited time, for children with DMD.
Official drug approvalDMD
USANew treatments and approvals
Prademagene zamikeracel receives FDA approval for wounds in butterfly skin disease (RDEB)
The FDA has approved prademagene zamikeracel for wounds in patients with recessive dystrophic epidermolysis bullosa.
Official drug approvalEB
EUNew treatments and approvals
Positive opinion in the EU for subcutaneous efgartigimod for CIDP
The European Medicines Agency's scientific committee, the CHMP, issued a positive opinion on April 25, 2025.
Official drug approvalCIDP
EUNew treatments and approvals
EU approves a gene therapy gel applied to wounds in dystrophic butterfly skin disease
The European Commission approved beremagene geperpavec for wounds in dystrophic butterfly skin disease (dystrophic epidermolysis bullosa).
Official drug approvalEB
USANew treatments and approvals
Fitusiran approved in the USA for hemophilia A and B
The FDA has approved fitusiran to prevent bleeding in patients with hemophilia A or B.
Official drug approvalHemophilia
USANew treatments and approvals
Diazoxide choline approved in the USA for excessive hunger in Prader-Willi syndrome
On March 26, 2025, the US Food and Drug Administration (FDA) approved diazoxide choline tablets for excessive hunger in Prader-Willi syndrome.
Official drug approvalPrader-Willi