Compassionate use - Medical need

The Royal Decree of 25 April 2014 (RD in French version) amending the Royal Decree of 14 December 2006 on medicinal products for human and veterinary use (RD in french version) was published in the Belgian Official Journal on 12 June 2014 and comes into effect on 1 July 2014.

All compassionate use programs and medical need programs submitted from that date must follow the procedure described in that text.

The FAMHP will request a contribution for each file submitted. The FAMHP requests no longer to make advance payments, but to wait for the invoice (or invitation to pay) with structured notice for the payment. More information about the new method can be found here. The amount of the contribution for Unmet Medical Need files can be found here. Please indicate in the cover letter to whom the invoice should be sent.

The guidance describing, among others, the process to submit a compassionate use program and medical need program is here available. You will also find below, the appendices of this new guidance :

  • CUP-UMN guidance
  • Annex I :     Royal Decree of 25 April 2014 amending the Royal Decree of 14 December 2006 (french and dutch version)
  • Annex II :    Application form to request a Compassionate Use Program or a Medical Need Program
  • Annex III :   Template of Compassionate Use Program protocol. New version 27/06/2023
  • Annex IV :   Summarized information for publication (EN-FR-NL)
  • Annex V :    Labeling
  • Annex VI :   Template of Medical Need Program protocol. New version 27/06/2023
  • Annex VII :  CUP Physician Declaration
  • Annex VIII : MNP Physician Declaration
  • Annex IX : e-submission through the CESP: this procedure must be followed exactly
  • Annex X : Application form re-evaluation

Please submit any specific questions via e-mail at umn@afmps-fagg.be.

FAQ

A list of frequently asked questions regarding the application for UMN can be found below and will be regularly updated :

FAQ

Authorised programs

Commercial name Active substance Indication Last update
Cibinqo abrocitinib the treatment of adolescents (12-17 years old) with severe atopic dermatitis or who have participated to the Abrocitinib Expanded access JADE REAL – B7451064 with EudraCT reference 2020-003610-12.  23/04/2024
Sotatercept  MK-7962 Adult patients with Pulmonary Arterial Hypertension (PAH)
 
09/04/2024
BALVERSA erdafitinib This compassionate use program (CUP) is intended to provide early access to erdafitinib for the adult patients with locally advanced unresectable or metastatic urothelial carcinoma (UC), harboring susceptible FGFR3 genetic alterations with disease progression during or following at least one line of programmed death PD-1 or PD-L1 inhibitor in the locally advanced unresectable or metastatic treatment setting. 03/04/2024
Fintepla for DS Fenfluramine (as fenfluramine hydrochloride) Fintepla for the treatment of patients with Dravet syndrome who completed the open label study ZX008-1900 (EP0215; EudraCT number 2019-001331-31), or the open label study titled “Fenfluramine als anti-epilepticum bij Dravet syndroom en epileptische encephalopathieën” (EudraCT number 2011-004114-42), or the open label study EP0213 (ClinicalTrials.gov ID number NCT06118255) and, in the opinion and the clinical judgement of the treating physician, would continue to benefit from a treatment with Fintepla 2.2 mg/ml oral solution (fenfluramine), which is not yet commercially available in Belgium. 03/04/2024
Fintepla for LGS Fenfluramine (as fenfluramine hydrochloride) Fintepla for the treatment of patients with Lennox-Gastaut syndrome who completed the open label study ZX008-1900 (EP0215, EudraCT number 2019-001331-31), or the open label study titled “Fenfluramine als anti-epilepticum bij Dravet syndroom en epileptische encephalopathieën” (EudraCT number 2011-004114-42) or the open label study FFA-LGS (EudraCT number 2015-004008-46) and, in the opinion and the clinical judgement of the treating physician, would continue to benefit from a treatment with Fintepla 2.2 mg/ml oral solution (fenfluramine), which is not yet commercially available in Belgium. 03/04/2024
Orladeyo® Berotralstat dihydrochloride Orladeyo is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 years and older.  13/02/2024
Olorofim Olorofim invasive fungal infections in patients lacking alternative treatment options. 18/01/2024
Jardiance (empagliflozin)  empagliflozin The use of Jardiance® in adults for the treatment of chronic kidney disease with an eGFR < 45 ml/min/1.73m² & uACR <200mg/g or with an eGFR ≥60 to <90 mL/min/1.73m2 in case of uACR ≥200 mg/g 20/06/2024
Omjjara Momelotinib  the treatment of disease related splenomegaly or symptoms in adult patients with moderate to severe anaemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis and who are Janus Kinase (JAK) inhibitor naïve or who have been treated with ruxolitinib.  07/02/2024
Jaypirca Pirtobrutinib treatment of Adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have been previously treated with a Bruton’s tyrosine kinase (BTK) inhibitor 24/11/2023
Talzenna talazoparib n combination with Xtandi (enzalutamide) for the treatment of adult patients with metastatic castration-resistant prostate cancer (mCRPC) in whom chemotherapy is not clinically indicated 27/11/2023
Camzyos Mavacamten The treatment of symptomatic (New York Heart Association, NYHA, class II III) obstructive hypertrophic cardiomyopathy (oHCM) in adult patients.     07/11/2023
Epcoritamab Epcoritamab Third line and subsequent lines of treatment (3rd +) Relapse/Refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL) 30/10/2023
Sparsentan Sparsentan Treatment in adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion > 1g/day 19/10/2023
Talquetamab Talquetamab As monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least 3 prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy 03/06/2024
Sunlenca Lenacapavir in combination with other antiretroviral(s), for the treatment of adults with multidrug resistant HIV-1 infection for whom it is otherwise not possible to construct a suppressive anti-viral regimen. 05/10/2023
Kaftrio Elexacaftor/Tezacaftor/Ivacaftor and Ivacaftor for Patients 2 Through 5 Years of Age With Cystic Fibrosis Who Have At Least One F508del Mutation in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene 21/02/2024
Jemperli dostarlimab in combination with carboplatin-paclitaxel as first-line treatment of adult patients with mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) primary advanced or recurrent endometrial cancer (EC) and who are candidates for systemic therapy 31/01/2024
Livmarli maralixibat chloride treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 2 months of age and older 20/06/2023
Nirogacestat Nirogacestat Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF) 15/03/2024

Rybrevant

amivantamab

treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who have epidermal growth factor receptor (EGFR) Exon 20 insertion (Exon20ins), and whose disease has progressed during or after current standard of care platinum-based chemotherapy.

04/04/2023

 

Hepcludex

bulevirtide

for the treatment of chronic hepatitis delta virus (HDV) infection in plasma (or serum) HDVRNA
positive adult patients with compensated liver disease

04/04/2023

 

Ebvallo

tabelecleucel

Monotherapy for the treatment of adult and paediatric patients 2 years of age and older with relapsed or refractory Epstein Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate.

26/06/2024

Ocaliva®

Obeticholic acid

Treatment of patients with Primary Biliary cholangitis who completed the Long-Term Safety Extension of the POISE phase 3 trial (747-301 / EudraCT 2011-004728-36)

08/03/2024

Myalepta

Metreleptine

Treatment of patients with generalized lipodystrophy or patients with partial lipodystrophy and uncontrollable metabolic disorders.

20/03/2023

 

Vamorolone

17α,21‐dihydroxy‐16α‐methyl‐pregna‐1,4,9(11)‐triene‐3,20‐dione

Duchenne Muscular Dystrophy in boys who are completing the VBP15-004 trial

16/03/2023

 

Tofersen

tofersen

Amyotrophic Lateral Sclerosis (ALS) Associated with a Mutation in the Superoxide Dismutase 1 (SOD1) Gene

03/07/2024

Pritelivir

Pritelivir

treatment of acyclovir-resistant mucocutaneous HSV (Herpes Simplex Virus) infections in immunocompromised patient

12/03/2024

Privigen

Human immunoglobuline

Treatment of bleeds in patients with acquired von Willebrand syndrome.

07/10/2022

Ozawade

pitolisant

to improve wakefulness and reduce excessive daytime sleepiness (EDS) in adult patients with obstructive sleep apnoea (OSA) whose EDS has not been satisfactorily treated by, or who have not tolerated, OSA primary therapy, such as continuous positive airway pressure (CPAP)

07/10/2022

Dupixent

dupilumab

add-on therapy with intranasal corticosteroids for treatment of severe Chronic Rhinosinusitis with Nasal Polyps (CRSwNP) and comorbid asthma patients who have participated and exited the phase 4 clinical study EVEREST (LPS16747)

22/12/2023

Bylvay

odevixibat

treatment of cholestatic liver disease in patients with Alagille Syndrome.

12/01/2024

Avacopan

Avacopan

in combination with a rituximab or cyclophosphamide regimen, is indicated for the treatment of adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA)

05/09/2022

Kanuma

sebelipase alfa

Treatment of paediatric and adult patients with lysosomal acid lipase deficiency (LAL-D) who participated to the open label phase 2 study (LAL-C06).

11/08/2022

Selumetinib

Selumetinib

neurofibromatosis type 1 with symptomatic, inoperable plexiform neurofibromas in paediatric patients with neurofibromatosis type 1 (NF1) aged ≥ 3 years and ≤ 18 years

11/08/2022

Scemblix

asciminib

treatment of Chronic Myelogenous Leukemia (CML) patients in chronic phase with a T315I mutation and previously treated with ≥2 TKIs

18/01/2024

Tolvaptan

Tolvaptan

Treatment of Children and Adolescent Patients until they reach 18 years of age with Autosomal Dominant Polycystic Kidney Disease Following Successful Participation and Completion in the 156-12-298 Clinical Trial

10/08/2022

Selinexor

selinexor

Adult patients with relapsed or refractory multiple myeloma (RRMM), who have have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy.

01/09/2023

Leriglitazone

Leriglitazone

Treatment of Friedreich’s Ataxia

09/08/2022

Velmanase alfa®

Velmanase alfa

Treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis

29/07/2022

Strensiq®

Asfotase alfa

long-term enzyme replacement therapy in patients with - hypophosphatasia (HPP) in whom the first symptoms presented before the age of 18, to treat the bone manifestations of the disease.

17/02/2020

Jakavi

Ruxolitinib

Treatment of Corticoid-refractory acute graft vs. host disease after allogeneic stem cell transplantation in pediatric patients <12 years old, who cannot be adequately treated with commercially available alternatives.

01/06/2023

Jakavi

Ruxolitinib

the treatment of Corticosteroid-refractory chronic graft vs. host disease after allogeneic stem cell transplantation, for pediatric patients younger than 12 years who cannot be adequately treated with commercially available alternatives.

22/06/2023

 

Translarna®

ataluren

Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, inambulatory patients aged 5 years and older for patients who have been treated with this medicinal product as part of the clinical trials (studies 019 and 020E) that are currently in the close-out process.

15/09/2017

 

On hold programs

Closed programs

Last updated on 03/07/2024