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
  • Annex IV :   Summarized information for publication (EN-FR-NL)
  • Annex V :    Labeling
  • Annex VI :   Template of Medical Need Program protocol
  • 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
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.

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.

iNOmax® iNOmax

patients who suffer from pulmonary hypertension associated with COPD and who participated in the PULSE-COPD-007 study

Velmanase alfa®

Velmanase alfa

Treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis
Jakavi Ruxolitinib Treatment of patients with corticoid-refractory chronic graft vs. host disease after allogeneic stem cell transplantation, who cannot be adequately treated with commercially available alternatives.
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)

Jakavi Ruxolitinib Treatment of patients with corticoid-refractory acute graft vs. host disease after allogeneic stem cell transplantation, who cannot be adequately treated with commercially available alternatives.
Privigen Human immunoglobuline Treatment of bleeds in patients with acquired von Willebrand syndrome.
Olorofim F901318 Treatment of invasive fungal infections due to Lomentospora prolificans, Scedosporium spp., Aspergillus spp., and other resistant fungi in patients lacking suitable alternative treatment.
Myalepta Metreleptine Treatment of patients with generalized lipodystrophy or patients with partial lipodystrophy and uncontrollable metabolic disorders.
Esbriet Pirfenidon Treatment of adult patients with unclassifiable interstitial lung disease (uILD) and who exit trial MA39189.
Esbriet pirfenidone treatment of patients with advanced idiopathic pulmonary fibrosis and risk of group 3 pulmonary hypertension (i.e. pulmonary hypertension occurring secondary to lung disease and/or hypoxia) and exiting study MA29957
Humira® adalimumab moderately to severely active Ulcerative Colitis in paediatric patients of 2 to 5 years old.
esketamine Esketamine ESK is intended for treatment‐resistant depression (Major Depressive Disorder in adults who have not responded to at least two different treatments with antidepressants in the current moderate to severe depressive episode). ESK must be given in combination with an SSRI or SNRI.
Vamorolone

Delta-1,4,9(11)-pregnatriene-17,21-dihydroxy-16-alpha-methyl-3,20-dione

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

Treatment of primary hyperoxaluria type 1 (PH1) in patients who have reached at least 37 weeks estimated gestational age (full‐term infant) at consent (or assent)

Fycompa

Perampanel

Seizures associated with Lennox Gastaut Syndrome (LGS) for pediatric patients under 12 years of age.

Forxiga® dapagliflozin Treatment of chronic kidney disease
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
Verquvo® vericiguat Adult patients with symptomatic chronic heart failure (New York Heart Association (NYHA) Class II-III-IV) with a reduced ejection fraction who are stabilized after a previous decompensation event requiring IV therapy.
Rinvoq® upadacitinib patients (adults and adolescents ≥ 12 years old) with severe atopic dermatitis who have no other suitable treatment option and are not eligible to participate in a clinical study
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.
Soliris® eculizumab patients with refractory generalized myasthenia gravis (rgMG) who completed the study ECU-MG-302 (Open label extension) and, in the opinion and the clinical judgement of the treating physician would continue to benefit from a treatment with Eculizumab (Soliris®) which is not yet commercially available for this indication in Belgium.
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.
Gilenya® fingolimod treatment of patients with relapsing-remitting multiple sclerosis (RRMS) who are included in medical need program CFTY720DBE02_C_IPS
Enhertu® trastuzumab deruxtecan adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens.
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).

Norditropin® somatotropin growth failure in children with achondroplasia/hypochondroplasia
Inrebic® fedratinib treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis have been treated with ruxolitinib.
Selumetinib Selumetinib neurofibromatosis type 1 with symptomatic, inoperable plexiform neurofibromas in paediatric patients with neurofibromatosis type 1 (NF1) aged ≥ 3 years and ≤ 18 years
Tebentafusp tebentafusp HLA-A*02:01 positive patients with metastatic uveal melanoma
Nexviadyme  avalglucosidase alfa treatment of adult Pompe disease patients who have participated and exit the clinical studies COMET (EFC14028) or NEO-EXT (LTS13769)
Xenpozyme olipudase alfa acid sphingomyelinase deficiency for patients who have participated and exit the clinical studies DFI-12712-ASCEND or LTS-13632
asciminib asciminib Patients with chronic myeloid leukemia (CML) in chronic phase previously treated with ≥ 2 TKIs

Tukysa®

tucatinib (ONT-380) in combination with trastuzumab and capecitabine for the treatment of adult patients with HER2 positive locally advanced or metastatic breast cancer who have received at least 2 prior anti HER2 treatment regimens.
tepotinib tepotinib Treatment of adult patients with advanced non-small cell lung cancer harboring mesenchymal-epithelial transition factor gene METexon14 (METex14) skipping alterations.
Leriglitazone Leriglitazone Treatment of Friedreich’s Ataxia
Darzalex daratumumab in combination with cyclophosphamide, bortezomib and dexamethasone for the treatment of adult patients with newly diagnosed systemic amyloid light chain (AL) amyloidosis
Tecartus autologous anti-CD19 CD3+ cells (CAR+ viable T cells) treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) after two or more lines of systemic therapy including a Bruton's tyrosine kinase (BTK) inhibitor
Otezla apremilast Treatment of adult patients with oral ulcers associated with Behçet’s disease (BD) who are candidates for systemic therapy. The patient has received prior treatment with a topical therapy AND with colchicine. The patient has shown to be resistant or intolerant to colchicine or has a documented contraindication against colchicine.
Evrenzo® roxadustat Treatment of symptomatic anaemia of Chronic Kidney Disease in non-dialysis and dialysis patients, and where use of available treatment (IV iron or injectable ESAs) is not appropriate or is expected not to be appropriate, or patients who have a known contraindication for the currently available treatment or who do not tolerate the currently available treatment, or are expected not to tolerate this product
Lenvima® lenvatinib in combination with pembrolizumab for the treatment of patients with advanced or recurrent endometrial carcinoma in adults who have disease progression on or following prior treatment with a platinum-containing therapy in any setting and who are not candidates for curative surgery or radiation. 
Jardiance® empagliflozin treatment of adult patients with heart failure with preserved ejection fraction (ejection fraction >40%)
Opdivo + Yervoy nivolumab + ipilimumab Treatment of adult patients with unresectable malignant pleural mesothelioma that have progressed after at least one line of treatment, including platinumbased doublet chemotherapy.
Trodelvy® sacituzumab govitecan treatment in monotherapy of adult patients with unresectable, locally advanced or metastatic triple-negative breast cancer (mTNBC) who have received two or more prior systemic therapies, including at least one of them for advanced disease and with no adequate treatment options
LUMYKRAS sotorasib Treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with KRAS G12C mutation and who have progressed after at least one prior line of systemic therapy
Kerendia finerenone Adult patients with chronic kidney disease (CKD) (stage 3 & 4 with albuminuria) associated with type 2 diabetes (T2D) who cannot be satisfactorily treated with the standard of care.
Aspaveli® pegcetacoplan Treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic despite treatment with a C5 inhibitor for at least 3 months.
Kaftrio ELX/TEZ/IVA and IVA alone Cystic fibrosis (CF) in patients from 6 to 11 years old who are homozygous for F508del (F/F genotype) or heterozygous for F508del and a minimal function (F/MF genotype) mutation.
Verzenios abemaciclib in combination with Endocrine Therapy as the adjuvant treatment of adult patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, nodepositive early breast cancer at high risk of recurrence
Vyxeos liposomal daunorubicin and cytarabine Newly diagnosed, treatment-related acute myeloid Leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC)
Pritelivir Pritelivir treatment of acyclovir-resistant mucocutaneous HSV (Herpes Simplex Virus) infections in immunocompromised patient
Adtralza  tralokinumab severe atopic dermatitis in adolescent patients (12- <18 years) who are candidates for systemic treatment (including patients coming from the Open Label Extension Study ECZTEND – LP0162-1337 with EudraCT reference 2018-000746-19)

On hold programs

Closed programs

Last updated on 08/08/2022