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 advfintepl
ance 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 |
| Mexiletine PR | Mexiletine | Mexiletine Prolonged Release (mexiletine-PR) for the treatment of adult Non-Dystrophic Myotonia | 26/06/2026 |
| Zepzelca | Lurbinectedin | The indication for the Compassionate Use Program (CUP) is: “Zepzelca (lurbinectedin), in combination with atezolizumab, is indicated for the maintenance treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC) whose disease has not progressed after first-line induction therapy with atezolizumab, carboplatin and etoposide”. | 01/07/2026 |
| Atrasentan | Atrasentan | Atrasentan is indicated for the treatment of adults with primary IgA nephropathy at risk of disease progression, with a urine protein excretion ≥1g/day and < 3.5g/day. | 25/06/2026 |
| Tibsovo | Ivosidenib | Monotherapy for the treatment of adult patients with acute myeloid leukemia (AML) and an isocitrate dehydrogenase-1 (IDH1) R132 mutation not responding to initial induction therapy or with relapsed disease after initial induction therapy who are no longer a candidate for intensive chemotherapy | 18/06/2026 |
| Jyseleca® | Filgotinib | Subjects with axial spondyloarthritis (axSpA) who have participated in the previous filgotinib clinical trial OLINGUITO (GLPG0634-CL-336) in Belgium and who, based on the clinical judgement of their treating physician, may benefit from continued treatment. |
26/05/2026 |
| Sparsentan | Sparsentan | Treatment in adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion > 1g/day | 20/04/2026 |
| CENRIFKI | Tolebrutinib | Tolebrutinib is indicated for the treatment of non-relapsing secondary progressive multiple sclerosis (nrSPMS) in adults. | 10/04/2026 |
| Zidesamtinib | Zidesamtinib | Treatment of patients with advanced ROS1 fusion-positive non-small cell lung cancer (ROS1+ NSCLC) who have previously received ≥ 1 prior ROS1 tyrosine kinase inhibitor (TKI) | 20/03/2026 |
| Itovebi | Inavolisib | Treatment of adult patients with PIK3CA-mutated, oestrogen receptor (ER)-positive, HER2-negative, locally advanced or metastatic breast cancer (LA/mBC) following recurrence on or within 12 months of completing adjuvant endocrine treatment. | 09/03/2026 |
| Fosmanogepix | Manogepix | Treatment of patients with Invasive Mold Infections (IMIs). | 11/12/2025 |
| Imfinzi | Durvalumab | Imfinzi® (durvalumab) in combination with FLOT chemotherapy as neoadjuvant and adjuvant treatment followed by adjuvant Imfinzi® monotherapy for the treatment of adults with resectable gastric or gastro-oesophageal junction adenocarcinoma (GC/GEJC). | 27/05/2026 |
| Pedmarqsi® | Sodium thiosulfate | Prevention of ototoxicity induced by cisplatin (CIS) chemotherapy in patients 1 month to < 18 years of age with localized, non-metastatic, solid tumors. | 14/08/2025 |
| Zynyz | Retifanlimab | first-line treatment of adult patients with metastatic or with inoperable locally recurrent Squamous Carcinoma of the Anal Canal (SCAC) with retifanlimab in combination with carboplatin and paclitaxel | 06/03/2026 |
| Finerenone | Finerenone | Chronic Kidney Disease in pediatric patients who participated in the FIONA open label extension study. | 25/03/2026 |
| Amvuttra | vutrisiran | treatment of patients with transthyretin-mediated amyloidosis (ATTR amyloidosis) with cardiomyopathy (ATTR-CM) | 27/10/2025 |
| plozasiran | plozasiran | Familial Chylomicronemia Syndrome (FSC) | 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. | 19/06/2026 |
| 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. | 25/06/2026 |
| 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 |
17/07/2025 |
| Duvyzat | Givinostat | Duvyzat is indicated for the treatment of Duchenne muscular dystrophy (DMD) in ambulant patients, aged 6 years and older, and with concomitant corticosteroid treatment. | 12/02/2026 |
| 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 | 29/05/2026 |
| Fintepla | Fenfluramine hydrochloride | treatment of patients with CDKL5 deficiency disorder who completed study ZX008-2103 (EP0216; EudraCT number 2021-003222-76) | 06/01/2026 |
| Voranigo | vorasidenib | treatment of predominantly non-enhancing grade 2 astrocytoma or oligodendroglioma 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 and are not in immediate need of radiotherapy or chemotherapy | 31/03/2026 |
| 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 |
07/07/2026 |
| Brukinsa | Zanubrutinib | monotherapy to adult patients with relapsing or refractory Mantle Cell Lymphoma (MCL) who have received at least 1 prior therapy. | 13/11/2025 |
| Bimzelx | Bimekizumab | Adults with moderate to severe Hidradenitis Suppurativa (HS) (acne inversa) | 29/10/2024 |
| Winrevair | Sotatercept (MK-7962) | Adult patients with Pulmonary Arterial Hypertension (PAH) with WHO Functional Class (FC) II/III or FC IV. | 10/09/2025 |
| 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 |
| Olorofim | Olorofim | invasive fungal infections in patients lacking alternative treatment options. | 18/01/2024 |
| Nirogacestat | Nirogacestat | treatment of Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF) | 02/06/2025 |
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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. |
27/10/2025
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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) |
07/01/2026 |
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Metreleptine |
as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: |
07/07/2026
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Pritelivir |
treatment of acyclovir-resistant mucocutaneous HSV (Herpes Simplex Virus) infections in immunocompromised patient |
29/08/2025 |
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Human immunoglobuline |
Treatment of bleeds in patients with acquired von Willebrand syndrome. |
07/10/2022 |
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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 |
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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 |
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asciminib |
treatment of Chronic Myelogenous Leukemia (CML) patients in chronic phase with a T315I mutation and previously treated with ≥2 TKIs |
28/11/2024 |
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Leriglitazone |
Treatment of Friedreich’s Ataxia |
09/08/2022 |
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Velmanase alfa |
Treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis |
24/06/2026 |