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 :
Authorised programs
| Commercial name | Active substance | Indication | Last update |
| Amvuttra | vutrisiran | treatment of patients with transthyretin-mediated amyloidosis (ATTR amyloidosis) with cardiomyopathy (ATTR-CM) | 25/04/2025 |
| plozasiran | plozasiran | Familial Chylomicronemia Syndrome (FSC) | 23/04/2025 |
| Jayprica® | pirtobrutinib | Pirtobrutinib for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), who have been exposed to a Bruton’s tyrosine kinase (BTK) inhibitor and venetoclax (i.e. double-exposed patients). | 23/04/2025 |
| Welireg® | Belzutifan | Belzutifan (Welireg®) is indicated for the treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for VHL associated, localized renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumours (pNET), and for whom localized procedures are unsuitable. | 22/04/2025 |
| Vyvgart | efgartigimod alfa | Medical Need Program with Vyvgart 1000 mg solution for subcutaneous injection as monotherapy for adult patients with progressive or relapsing active Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP), after prior treatment with immunoglobulins and/or corticosteroids. | 18/04/2025 |
| Kisqali | Ribociclib | The adjuvant treatment of patients with ribociclib diagnosed with early-stage Hormone Receptor-positive (HR+), Human Epidermal growth factor Receptor 2-negative (HER2─) breast cancer (stages II and III) requiring adjuvant treatment with an endocrine treatment based regimen. | 17/04/2025 |
| Blenrep | Belantamab mafodotin | Belantamab mafodotin is indicated in adults for the treatment of relapsed or refractory multiple myeloma: • in combination with bortezomib and dexamethasone in patients who have had at least one prior therapy; and • in combination with pomalidomide and dexamethasone in patients who have received at least one prior therapy including lenalidomide |
14/04/2025 |
| Duvyzat | Givinostat | Givinostat Compassionate Use Programme (CUP) for treatment of ambulant patients aged 6 years and older with Duchenne Muscular Dystrophy (DMD) | 11/04/2025 |
| Teplizumab | Teplizumab | Teplizumab is indicated for the treatment of adult and pediatric patients aged 8 years and older with stage 2 type 1 diabetes to delay the onset of stage 3 type 1 diabetes Teplizumab is administered by intravenous infusion (over a minimum of 30 minutes), using a body surface area-based dosing, once daily for 14 consecutive days as follows: • Day 1: 65 μg/m2 • Day 2: 125 μg/m2 • Day 3: 250 μg/m2 • Day 4: 500 μg/m2 • Days 5 through 14: 1030 μg/m2 Should a scheduled teplizumab infusion be missed, treatment shall be continued on the next scheduled day and any remaining doses be administered on subsequent days to complete the 14-day treatment. Two doses must not be administered on the same day. Teplizumab must be diluted prior to administration. |
02/04/2025 |
| Aspaveli | Pegcetacoplan | Treatment of primary immune complex and C3-mediated glomerulopathy | 21/03/2025 |
| Fintepla | Fenfluramine hydrochloride | treatment of patients with CDKL5 deficiency disorder who completed study ZX008-2103 (EP0216; EudraCT number 2021-003222-76) | 18/03/2025 |
| ELAHERE | mirvetuximab soravtansine | Treatment in monotherapy of adult patients with folate receptor-alpha (FRα) positive, platinum-resistant high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received one to three prior systemic treatment regimens. | 26/02/2025 |
| Vorasidenib | vorasidenib | the treatment of predominantly non-enhancing grade 2 astrocytoma or oligodendroglioma (as per World Health Organization (WHO) 2021 criteria) with an IDH1 R132 or IDH2 R172 mutation in adult and adolescent patients aged 12 years and older and weighing >40kg who only had surgical intervention including biopsy, subtotal resection or gross total resection and are not in immediate need of radiotherapy or chemotherapy | 31/01/2025 |
| TAGRISSO® |
Osimertinib | For the treatment of adult patients with locally advanced, unresectable NSCLC whose tumours have EGFR exon 19 deletions or exon 21 (L858R) substitution mutations and whose disease has not progressed during or following platinum-based chemoradiation therapy. | 22/01/2025 |
| Xtandi | enzalutamide | As monotherapy or in combination with androgen deprivation therapy for the treatment of adult men with high risk biochemical recurrent (BCR) non-metastatic hormone sensitive prostate cancer (nmHSPC) who are unsuitable for salvage radiotherapy | 19/02/2025 |
| Belumosudil | Belumosudil | chronic Graft Versus Host Disease (cGVHD) after failure of at least 2 prior lines of systemic therapy | 07/11/2024 |
| Vyvgart | efgartigimod alfa | Acetylcholine receptor antibody negative patients with gMG who participated in and exited study ARGX-113-2002 OR who were treated via early access | 29/10/2024 |
| Orserdu | elacestrant | Treatment of postmenopausal women, and men, with estrogen receptor (ER)-positive, HER2-negative, locally advanced, or metastatic breast cancer with an activating ESR1 mutation, who have disease progression following one or two lines of endocrine therapy including a CDK 4/6 inhibitor. | 11/10/2024 |
| Brukinsa | Zanubrutinib | monotherapy to adult patients with relapsing or refractory Mantle Cell Lymphoma (MCL) who have received at least 1 prior therapy. | 30/09/2024 |
| Ultomiris | Ravulizumab | treatment of adult patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) who are anti-aquaporin 4 antibody-positive (AQP4 +) | 17/09/2024 |
| Bimzelx | Bimekizumab | Adults with moderate to severe Hidradenitis Suppurativa (HS) (acne inversa) | 29/10/2024 |
| Lynparza | Olaparib | Lynparza® (olaparib) in combination with durvalumab for the maintenance treatment of adult patients with primary advanced or recurrent endometrial cancer (EC) that is mismatch repair proficient (pMMR) whose disease has not progressed on first-line treatment with durvalumab in combination with carboplatin and paclitaxel. | |
| 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 |
| Winrevair | sotatercept | Adult patients with Pulmonary Arterial Hypertension (PAH) with WHO Functional Class (FC) II/III”. |
03/12/2024 |
| 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. | 06/06/2025 |
| 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. | 06/06/2025 |
| Olorofim | Olorofim | invasive fungal infections in patients lacking alternative treatment options. | 18/01/2024 |
| 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 | 30/04/2025 |
| Sparsentan | Sparsentan | Treatment in adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion > 1g/day | 19/10/2023 |
| 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 |
| Nirogacestat | Nirogacestat | treatment of Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF) | 02/06/2025 |
|
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
|
|
|
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 |
|
|
Metreleptine |
Treatment of patients with generalized lipodystrophy or patients with partial lipodystrophy and uncontrollable metabolic disorders. |
20/03/2023
|
|
|
tofersen |
Amyotrophic Lateral Sclerosis (ALS) Associated with a Mutation in the Superoxide Dismutase 1 (SOD1) Gene |
27/05/2025 |
|
|
Pritelivir |
treatment of acyclovir-resistant mucocutaneous HSV (Herpes Simplex Virus) infections in immunocompromised patient |
12/03/2024 |
|
|
Human immunoglobuline |
Treatment of bleeds in patients with acquired von Willebrand syndrome. |
07/10/2022 |
|
|
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 |
|
|
odevixibat |
treatment of cholestatic liver disease in patients with Alagille Syndrome. |
12/01/2024 |
|
|
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 |
|
|
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 |
|
|
asciminib |
treatment of Chronic Myelogenous Leukemia (CML) patients in chronic phase with a T315I mutation and previously treated with ≥2 TKIs |
28/11/2024 |
|
|
Leriglitazone |
Treatment of Friedreich’s Ataxia |
09/08/2022 |
|
|
Velmanase alfa |
Treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis |
29/07/2022 |
|
|
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 |
|
|
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
|