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 |
| Trodelvy | sacituzumab govitecan |
as monotherapy is indicated for the treatment of adult patients with unresectable or metastatic hormone receptor (HR)-positive, HER2-negative breast cancer who have received endocrine-based therapy, and at least two additional systemic therapies in the advanced setting. |
08/11/2024 |
| Belumosudil | Belumosudil | chronic Graft Versus Host Disease (cGVHD) after failure of at least 2 prior lines of systemic therapy | 07/11/2024 |
| 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. | 05/11/2024 |
| Imfinzi | durvalumab | Imfinzi® (durvalumab) as monotherapy for the treatment of patients with limited stage small-cell lung cancer (LS-SCLC) who have not progressed following platinum-based chemoradiation therapy. | 31/10/2024 |
| 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 |
| Keytruda | Pembrolizumab | for the adjuvant treatment of renal cell carcinoma patients with intermediate-high risk and high risk of recurrence after nephrectomy. | 02/10/2024 |
| 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 |
| Keytruda | Pembrolizumab | in combination with chemoradiotherapy (external beam radiation therapy followed by brachytherapy) for the treatment of locally advanced FIGO 2014 stage III-IVA cervical cancer, in adults who have not received prior definitive therapy. | 13/09/2024 |
| Altuvoct | Efanesoctocog alfa | Treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency). | 10/09/2024 |
| Bimzelx | Bimekizumab | Adults with moderate to severe Hidradenitis Suppurativa (HS) (acne inversa) | 29/10/2024 |
| Rybrevant | Amivantamab | in combination with carboplatin and pemetrexed for the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with activating EGFR Exon 20 insertion mutations | 12/08/2024 |
| Alecensa | alectinib | Alecensa as monotherapy is indicated as adjuvant treatment following complete tumour resection for adult patients with ALK-positive NSCLC at high risk of recurrence (see selection criteria). | 19/07/2024 |
| 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. | 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 |
| Olorofim | Olorofim | invasive fungal infections in patients lacking alternative treatment options. | 18/01/2024 |
| 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 | 23/09/2024 |
| 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 | 27/11/2023 |
| 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 | Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF) | 15/03/2024 |
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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. |
04/04/2023
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bulevirtide |
for the treatment of chronic hepatitis delta virus (HDV) infection in plasma (or serum) HDVRNA |
04/04/2023
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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) |
08/03/2024 |
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Metreleptine |
Treatment of patients with generalized lipodystrophy or patients with partial lipodystrophy and uncontrollable metabolic disorders. |
20/03/2023
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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
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tofersen |
Amyotrophic Lateral Sclerosis (ALS) Associated with a Mutation in the Superoxide Dismutase 1 (SOD1) Gene |
03/07/2024 |
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Pritelivir |
treatment of acyclovir-resistant mucocutaneous HSV (Herpes Simplex Virus) infections in immunocompromised patient |
12/03/2024 |
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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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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 |
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odevixibat |
treatment of cholestatic liver disease in patients with Alagille Syndrome. |
12/01/2024 |
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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 |
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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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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 |
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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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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 |
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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 |
29/07/2022 |
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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 |
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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
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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 |