Sanofi / Genzyme

The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and non-research related purposes. The objectives of the Registry are: * To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention. * To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care. * To characterize the Pompe disease population. * To evaluate the long-term effectiveness of alglucosidase alfa.Show more

This is a parallel group, Phase 2, 2-arm study to measure the treatment effect and safety of rilzabrutinib dose 1 or rilzabrutinib dose 2 in participants with Graves' disease, with and without Graves' orbitopathy, aged 18 years or older. Study details include: * Screening period (up to 4 weeks). * Treatment period (up to 16 weeks). * Follow-up period (4 weeks). The number of visits will be up to 13.Show more

This is a parallel, Phase 3, two-arm, open-label study to evaluate the efficacy and safety of treatment with fitusiran prophylaxis administered to male pediatric participants (aged 1 to \<12 years) who have severe hemophilia A or B, with or without inhibitory antibodies to FVIII or FIX. Number of participants: Approximately 85 participants will be enrolled into the study: * Approximately 60 fitusiran-naïve participants with severe hemophilia A or B, with or without inhibitors (fitusiran-naïve arm), and * Approximately 25 participants with severe hemophilia A or B with inhibitors rolling over from the EFC15467\* dose confirmation study (roll-over arm). * Fitusiran has been investigated in the pediatric population in study EFC15467, which enrolled male participants aged 1 to \<12 years with hemophilia A or B with inhibitors to examine the safety and tolerability of fitusiran in the pediatric population. Participants will be enrolled into 1 of 2 arms: * Fitusiran-naïve: these participants have not previously received fitusiran, and they will undergo screening and study eligibility assessments. Once enrolled, they will go through a 24-week standard of care (SOC) period before starting fitusiran prophylaxis. * Roll-over participants from the EFC15467 study: only participants who are still on active treatment in study EFC15467 and consenting to study EFC17905 will be eligible to roll over. They will not need to undergo screening or further eligibility assessments. They will directly enroll into the fitusiran treatment period and continue treatment on their current fitusiran dose. The duration of fitusiran treatment will be up to 160 weeks for the fitusiran-naïve arm and up to 60 weeks for the roll-over arm.Show more

Historically, the frailest patients with multiple myeloma are under-represented in clinical trials, and have very high rates of treatment discontinuation, and early treatment mortality. The investigators hypothesize that a go-slow gentle approach to starting treatment in such patients, starting with just Isatuximab and dexamethasone with a gentle introduction to lenalidomide third cycle onwards, may improve treatment adherence and quality of life. The goal of this clinical trial is to learn if a go-slow approach to treating MM in ultra-frail patients may improve the ability to adhere to treatment and improve quality of life.Show more

This is a Phase 3, parallel group, 2-arm, randomized, double blind, placebo-controlled, 52-week treatment study to assess the efficacy and safety of rilzabrutinib as a treatment for adult patients with active IgG4-RD. The purpose of this study is to measure time to adjudicated IgG4-RD clinical disease flare, and other relevant efficacy endpoints including flare-free rate, control of IgG4-RD disease activity, use of GC rescue and safety parameters such as treatment-emergent adverse events, clinical laboratory values and electrocardiograms (ECG) in participants aged 18 years and above, diagnosed with IgG4-RD and treated with rilzabrutinib tablets over a 52-week placebo-controlled period. Study details include: The study duration will be up to 60 weeks, including a Screening period of 4 to 6 weeks, a 52-week double blind treatment period, and 2 weeks of follow up (plus an optional OLE of 108 weeks). The number of visits will be 16 (plus an optional 9 visits during the OLE).Show more

This is a parallel-group, Phase 3, double-blind, 2-arm study to investigate the efficacy, safety, PK and PD of oral rilzabrutinib in achieving durable Hb response (DHR) compared with placebo in approximately 90 male and female participants ≥ 18 years of age with a confirmed diagnosis of primary wAIHA. Following a 4-week screening period, eligible participants will be randomized in a 2:1 ratio to receive rilzabrutinib or placebo in primary analysis period (PAP) for a duration of up to 24 weeks. All participants who completed PAP will then continue in open-label period (OLP) to receive rilzabrutinib for a duration of 28 weeks. Upon the completion of OLP, only participants who demonstrate Hb increase during the last 8 weeks of OLP per specified criteria in the protocol will be eligible to continue in long-term extension (LTE) of the study. The duration of the LTE period will be from the first-participant-in (FPI)-LTE until the last participant completes 52 weeks in LTE. The safety follow-up period of this study following treatment completion or discontinuation will be 2 weeks.Show more

This is a sequential Phase 1/2, two-part, multicenter study on safety, tolerability, and efficacy of one-time intravitreal SAR446597 for the treatment of participants with Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD). The core phase duration will be approximately 2 years for each participant. An Extended Follow-Up (EFU) phase of 3 years follows the core phase. The treatment is a one-time intravitreal injection of SAR446597 (or sham as applicable in Part II).Show more

To learn if isatuximab can help to control highrisk MM when given in combination with lenalidomide after an autologous stem cell transplantation (ASCT).

Sanofi / Genzyme has 20 FDA-approved drugs with a rare disease use that we track, as of November 2026.

Xenpozyme (olipudase alfa-rpcp), approved 2022: Niemann-Pick Disease.

Wayrilz (rilzabrutinib), approved 2025: Immune Thrombocytopenia.

Aubagio (teriflunomide), approved 2012: Multiple Sclerosis.

Cablivi (caplacizumab), approved 2019: Thrombotic Thrombocytopenic Purpura.

Qfitlia (fitusiran), approved 2025: Hemophilia A, Hemophilia B.

Altuviiio (efanesoctocog alfa), approved 2023: Hemophilia A.

Fabrazyme (agalsidase beta), approved 2003: Fabry Disease.

Cerdelga (eliglustat), approved 2014: Gaucher Disease.

And 12 more: see the FDA-approved drugs section on this page.

Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.

Yes, for 14 of its FDA-approved rare disease medicines that we track, each checked on Sanofi / Genzyme's own website.

Xenpozyme: Sanofi CareConnect Personalized Support Services, 1-800-745-4447. Offers copay help with private insurance and a free medicine program. Checked November 9, 2026.

Wayrilz: HemAssist Sanofi Support, 1-833-723-5463. Offers copay help with private insurance, a free medicine program and insurance help. Checked September 29, 2026.

Aubagio: MS One to One (AUBAGIO Co-Pay Program and One Start). Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked September 24, 2026.

Cablivi: Sanofi HemAssist, 1-833-723-5463. Offers copay help with private insurance, a free medicine program and insurance help. Checked November 2, 2026.

Qfitlia: Sanofi HemAssist, 1-833-723-5463. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked September 24, 2026.

Altuviiio: Sanofi HemAssist, 1-833-723-5463. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked October 17, 2026.

The other 8 are on the help paying page.

Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page.

Eligibility is decided by each program, so call the number listed before a prescription is filled.