QALSODY tofersen 100 mg/15mL Injection
🆔 Identity & classification
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🏷️ RxNorm drug class
This medicine belongs to the Antisense Oligonucleotide class.
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🏭 Manufacturer & labeler
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🩺 Clinical
Tofersen injection is used to treat a certain type of amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease; a condition in which the nerves that control muscle movement slowly die, causing the muscles to shrink and weaken) in adults with a certain genetic make-up. Tofersen injection is in a class of medications called antisense oligonucleotides. It works by decreasing the production of a harmful protein that builds up in the brain and spinal cord in ALS patients.
Read the full MedlinePlus article ↗Patient education
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🧪 Inactive Ingredients / Excipients
Inactive ingredients, also called excipients, are components of the drug product other than the active ingredient. They may include fillers, dyes, coatings, preservatives, flavors, or other formulation ingredients.
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UNII M4I0D6VV5M
Calcium chloride is a salt compound that acts as a firming agent and source of calcium ions in medications. It's used in formulations to help maintain tablet structure, improve texture, or serve as a buffering agent.
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UNII 02F3473H9O
A mineral salt used in medications as a source of magnesium. It helps bind ingredients together, improve texture, and maintain the product's stability during storage.
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UNII 660YQ98I10
Potassium chloride is a mineral salt used in medicines as a buffer and to help maintain the proper pH balance and stability of the formulation during storage and use.
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UNII 451W47IQ8X
Sodium chloride is common table salt. It's used in medicines as a buffer to maintain proper pH, as a filler to add bulk, or to adjust the osmotic balance in liquid formulations.
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UNII GR686LBA74
Sodium phosphate, dibasic is a salt derived from phosphoric acid. It acts as a buffer to help maintain the medicine's pH balance and may serve as a binder or filler in tablets and capsules.
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UNII 3980JIH2SW
A salt form of phosphoric acid that acts as a buffer and pH adjuster in medicines. It helps keep the product at the correct acidity level for stability and effectiveness.
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UNII 059QF0KO0R
Water is a liquid solvent that dissolves and mixes ingredients together in liquid medicines, syrups, and injections. It helps distribute the active drug evenly throughout the product.
7 inactive ingredients listed in the exact product block matched to this NDC.
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ingredient classCode="IACT" elements from the exact product block matched by this NDC. Label-section narrative from DailyMed / the openFDA label index is shown separately when available.Inactive ingredient FAQ
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💲 Pricing
A drug doesn't have one price. Each row is a different public payment system, and none is what you'd pay at the counter — that depends on your insurance. The ⓘ on each row explains what it measures.
| Price system | Per each | Per package |
|---|---|---|
| Retail pharmacies payNADAC · weekly | Not in the retail survey — common for institutional, discontinued, or low-volume packs. | |
| Medicaid paysCMS SDUD · 12 mo | No recent Medicaid claims on file for this NDC — rare and low-volume NDCs are suppressed in the public data. | |
| Medicare drug plans payPart D · quarterly | No Part D plan price is available for this NDC in our data. | |
| Medicare Part B allowsASP · J1304 | $164.816 / J1304 unit | — |
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🧾 Billing & reimbursement
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🔁 Therapeutic equivalents
| Product | Labeler | Pack | NADAC/unit | TE | Status | Price vs. this |
|---|---|---|---|---|---|---|
| Qalsody 100 mg/15mLthis 64406-0109-01 | Biogen | 1 vial | — | — | FDA listed | — |
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⏳ Availability & generic status
We did not find an FDA-approved generic match for this exact strength, form and route. Patent/protection dates below may affect future generic timing.
Why the date isn’t exact: Generic timing can change because patents may be challenged, settled, licensed, added, removed, or worked around with a narrower label — and FDA approval does not always mean a pharmacy can get the generic today.
🛈 What do these terms mean?
- Patent
- Legal protection listed in the Orange Book that may delay generic approval or launch. Issued by the U.S. Patent & Trademark Office.
- Substance patent
- Covers the active drug molecule itself — the hardest to design around. A generic generally can’t launch until it expires.
- Formulation (product) patent
- Covers a specific formulation or dosage form. A generic can sometimes work around it with a different formulation.
- Method-of-use patent
- A patent covering one specific approved use of the drug — not necessarily the whole molecule. A generic can sometimes launch with a “skinny label” that carves out the protected use and keeps the others.
- Skinny label
- A generic label that omits a still-patented use when the FDA allows it — letting a generic reach the market for the unprotected uses.
- Exclusivity
- FDA-granted marketing protection, separate from patents — e.g. 5-yr new chemical entity, 7-yr orphan drug, or a +6-month pediatric extension.
- Paragraph IV
- A generic applicant’s formal challenge to a listed patent. It can potentially lead to earlier generic entry, but often involves litigation or a settlement.
- RLD / RS
- Reference Listed Drug — the brand product the FDA uses as the reference for generic applications. Reference Standard — the product the FDA expects generics to compare against in bioequivalence testing.
- TE / AB rating
- FDA therapeutic-equivalence rating. An AB rating generally means the FDA considers a generic therapeutically equivalent to — and substitutable for — the brand.
- LOE (loss of exclusivity)
- The latest patent or exclusivity currently listed — the loss-of-exclusivity / latest-listed-protection date shown on this page. Paragraph-IV challenges and settlements can move the real date earlier; FDA approval and a manufacturer’s decision to market can move it later.
Built from the FDA Orange Book. The bars above are scaled to each protection’s expiry; the red LOE marker is the last one to lapse.
| Patent | Type | Use code | Expires |
|---|---|---|---|
| US 10968453 ↗ | Method of use | U-3575 | Apr 1, 2035 |
| US 10669546 ↗ | Method of use | U-3575 | Apr 1, 2035 |
| US 10385341 ↗ | Drug substance | — | Apr 1, 2035 |
| Code | What it grants | Expires |
|---|---|---|
| NCE | New Chemical Entity (5-year) | Apr 25, 2028 |
| ODE-432 | Orphan Drug Exclusivity (7-year) | Apr 25, 2030 |
Is there a generic version of QALSODY 100 MG/15 ML VIAL?
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📊 Medicare Part D spend CMS · PART D · 2026 (Q1)
🔬 Reported adverse events (FAERS)
Top reported reactions
Age at onset
Reporter sex
Serious outcomes
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📦 Packaging — all sizes for this product
| Package NDC | Description | Marketing start | Status |
|---|---|---|---|
| 64406-0109-01 You're viewing this | 1 VIAL, SINGLE-USE in 1 CARTON (64406-109-01) / 15 mL in 1 VIAL, SINGLE-USE | 2023-04-25 | Active |
🧭 About this NDC listing & data coverage
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| NDC identity (package / product / labeler codes) | ✓ Available |
| Labeler | ✓ Available |
| Product & package description | ✓ Available |
| Marketing category & status | ✓ Available |
| Active ingredient / dosage form / route | ✓ Available |
| FDA label (SPL via DailyMed) | ✓ Available |
| Package photos | ✓ Available |
| Inactive ingredients (structured) | ✓ Available |
| NADAC pharmacy acquisition price (CMS) | — Not published for this NDC CMS publishes NADAC only for NDCs reported in its retail-pharmacy survey. |
| Orange Book / therapeutic-equivalence data | ✓ Available |
| HCPCS J-code billing crosswalk | ✓ Available |
| Medicaid utilization (CMS SDUD) | — Not published for this NDC CMS reports utilization only for NDCs with Medicaid claims above its privacy threshold. |
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📄 Full prescribing information FDA SPL
🎯 Indications and Usage ▾
1 INDICATIONS AND USAGE QALSODY is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 ( SOD1 ) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY [see Clinical Studies ( 14 )] . Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).
QALSODY is an antisense oligonucleotide indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 ( SOD1 ) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain observed in patients treated with QALSODY. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).
( 1 )
⏱️ Dosage and Administration ▾
2 DOSAGE AND ADMINISTRATION QALSODY is administered intrathecally ( 2.1 ) Dosing Information ( 2.1 ) Recommended dose: 100 milligrams (15 mL) per administration Initiate QALSODY treatment with 3 loading doses administered at 14day intervals. A maintenance dose should be administered once every 28 days thereafter. Preparation and Administration Instructions ( 2.2 ) Allow to warm to room temperature prior to administration Administer within 4 hours of removal from vial Prior to administration, remove approximately 10 mL of cerebrospinal fluid Administer as an intrathecal bolus injection over 1 to 3 minutes 2.1.
Dosing Information Recommended Dosage Administer QALSODY intrathecally using a lumbar puncture by, or under the direction of, healthcare professionals experienced in performing lumbar punctures. The recommended dosage is 100 mg (15 mL) of QALSODY per administration. Initiate QALSODY treatment with three (3) loading doses administered at 14-day intervals.
Administer a maintenance dose every 28 days thereafter. Missed Dose If the second loading dose is missed, administer QALSODY as soon as possible, and administer the third loading dose 14 days later. If the third loading dose or a maintenance dose is missed, administer QALSODY as soon as possible, and administer the next dose 28 days later.
2.2. Preparation and Administration Instructions Use aseptic technique when preparing and administering QALSODY intrathecally. Prepare and administer QALSODY according to the following steps: Preparation Vial preparation instructions Allow refrigerated QALSODY vial to warm to room temperature (25°C/77°F) prior to administration without using external heat sources [see Storage and Handling ( 16.2 )] .
Inspect the solution in the QALSODY vial prior to administration. Do not administer if particles are observed or the liquid in the vial is not clear and colorless to slightly yellow. Do not shake the QALSODY vial.
Procedural preparation instructions If indicated by the clinical condition of the patient, consider sedation. If indicated by the clinical condition of the patient, consider imaging to guide intrathecal administration of QALSODY. Prior to removing the vial's cap on the aluminum overseal, confirm readiness of the patient.
An unopened QALSODY vial can be returned to the refrigerator [see Storage and Handling ( 16.2 )] . Evaluate patients prior to and after intrathecal injection for the presence of potential conditions related to lumbar puncture, to avoid serious procedural complications. Administration Prior to administration, remove approximately 10 mL of cerebrospinal spinal fluid (CSF) using a lumbar puncture needle.
Prior to administration, remove the plastic cap and attach a needle to the syringe, for the purpose of withdrawing QALSODY from the vial. Insert the needle into the vial through the center of the overseal and withdraw the required dose of 15 mL (equivalent to 100 mg) from the vial. Do not dilute QALSODY.
External filters are not required. Administer QALSODY using a lumbar puncture needle as an intrathecal bolus injection over 1 to 3 minutes. QALSODY contains no preservatives.
Once drawn into the syringe, the solution should be administered immediately (within 4 hours of removal from the vial) at room temperature; otherwise, it must be discarded. Any unused contents of the single-dose vial should be discarded.
💊 Dosage Forms and Strengths ▾
3 DOSAGE FORMS AND STRENGTHS Injection: 100 mg/15 mL (6.7 mg/mL) as a clear and colorless to slightly yellow solution in a single-dose vial. Injection: 100 mg/15 mL (6.7 mg/mL) solution in a single-dose vial ( 3 )
⛔ Contraindications ▾
4 CONTRAINDICATIONS None. None ( 4 )
⚠️ Warnings and Cautions ▾
5 WARNINGS AND PRECAUTIONS Myelitis and/ or Radiculitis: Serious events of myelitis and radiculitis have been reported. Monitor for symptoms; diagnostic workup and treatment should be initiated according to the standard of care. ( 5.1 ) Papilledema and Elevated Intracranial Pressure: Serious events of papilledema and elevated intracranial pressure have been reported.
Monitor for symptoms; diagnostic workup and treatment should be initiated according to standard of care. ( 5.2 ) Aseptic Meningitis: Serious events of aseptic meningitis have been reported. Monitor for symptoms; diagnostic workup and treatment should be initiated according to standard of care.
( 5.3 ) 5.1. Myelitis and/or Radiculitis Serious adverse reactions of myelitis and radiculitis have been reported in patients treated with QALSODY. Six patients treated with QALSODY experienced myelitis or radiculitis in the clinical studies.
Two patients discontinued treatment with QALSODY and required symptomatic management with full resolution of symptoms. In the remaining 4 patients, symptoms resolved without discontinuation of QALSODY. If symptoms consistent with myelitis or radiculitis develop, diagnostic workup and treatment should be initiated according to the standard of care.
Management may require interruption or discontinuation of QALSODY. 5.2. Papilledema and Elevated Intracranial Pressure Serious adverse reactions of papilledema and elevated intracranial pressure have been reported in patients treated with QALSODY.
Four patients developed elevated intracranial pressure and/or papilledema. All patients received treatment with standard of care with resolution of symptoms, and no events led to discontinuation of QALSODY. If symptoms consistent with papilledema or elevated intracranial pressure develop, diagnostic workup and treatment should be initiated according to the standard of care.
5.3. Aseptic Meningitis Serious adverse reactions of aseptic meningitis (also called chemical meningitis or drug-induced aseptic meningitis) have been reported in patients treated with QALSODY. One patient experienced a serious adverse reaction of chemical meningitis, which led to discontinuation of QALSODY.
One patient experienced a serious adverse reaction of aseptic meningitis, which did not lead to discontinuation of QALSODY. In addition, nonserious adverse drug reactions of CSF white blood cell increased, and CSF protein increased have also been reported with QALSODY [see Adverse Reactions ( 6.1 )]. If symptoms consistent with aseptic meningitis develop, diagnostic workup and treatment should be initiated according to the standard of care.
🤒 Adverse Reactions ▾
6 ADVERSE REACTIONS The following clinically significant adverse reactions are discussed elsewhere in the labeling: Myelitis and/or Radiculitis [see Warnings and Precautions ( 5.1 )] Papilledema and Elevated Intracranial Pressure [see Warnings and Precautions ( 5.2 )] Aseptic Meningitis [see Warnings and Precautions ( 5.3 )] The most common adverse reactions ( ≥ 10% of patients treated with QALSODY and greater than placebo) were pain, fatigue, arthralgia, cerebrospinal fluid white blood cell increased, and myalgia. (Section 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Biogen at 1-877-725-7639 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
6.1. Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of QALSODY cannot be directly compared to rates in clinical trials of other drugs and may not reflect the rates observed in practice. The safety of QALSODY 100 mg was evaluated in 147 patients with SOD1-ALS.
The median patient exposure was 119.4 weeks (range from 4 to 212 weeks). QALSODY was evaluated in the placebo-controlled Study 1 and in the open label extension Study 2. In Study 1 Part C, approximately 43% were female; 57% were male; 64% were White and 8% were Asian.
The mean age at entry in Study 1 Part C was 49.8 years (range from 23 to 78 years). The most common adverse reactions (≥ 10% of patients treated with QALSODY and greater than placebo) were pain, fatigue, arthralgia, CSF white blood cell increased, and myalgia. Table 1 shows the common adverse reactions that occurred in at least 5% of patients treated with QALSODY and at a 5% or higher frequency than placebo.
Table 1: Adverse Drug Reactions That Occurred in At Least 5% of Patients Treated with QALSODY and at >5% Higher Frequency Than Placebo ‡‡ Pain includes preferred terms of pain, back pain, and pain in extremity. * CSF white blood cell increased includes preferred terms of CSF white blood cell increased and pleocytosis. Adverse Reaction Study 1 Part C QALSODY 100 mg (n = 72) % Placebo (n = 36) % Pain ‡‡ 42 22 Fatigue 17 6 Arthralgia 14 6 CSF white blood cell increased * 14 0 Myalgia 14 6 CSF protein increased 8 3 Musculoskeletal stiffness 6 0 Neuralgia 6 0 Less Common Adverse Reactions Serious adverse reactions of myelitis and radiculitis; papilledema and elevated intracranial pressure; and aseptic meningitis have occurred in patients treated with QALSODY [see Warnings and Precautions ( 5.1 , 5.2 , 5.3 )] .
In the long-term extension study, nonserious adverse reactions of pyrexia have occurred with repeat administration of QALSODY.
👥 Use in Specific Populations ▾
8 USE IN SPECIFIC POPULATIONS 8.1. Pregnancy Risk Summary There are no adequate data on developmental risks associated with the use of QALSODY in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively.
The background risk of major birth defects and miscarriage for the indicated population is unknown. Data Animal Data Subcutaneous administration of tofersen (0, 3, 10, 30 mg/kg) every other day to pregnant mice during the period of organogenesis resulted in no adverse effects on embryofetal development. Plasma exposure at the highest dose tested (30 mg/kg) was approximately 4 times that in humans at the recommended human dose (RHD) of 100 mg.
Subcutaneous administration of tofersen (0, 3, 10, 30 mg/kg) every other day to pregnant rabbits during the period of organogenesis resulted in no adverse effects on embryofetal development. Plasma exposure at the highest dose tested (30 mg/kg) was approximately 20 times that in humans at the RHD. Subcutaneous administration of tofersen (0, 3, 10, or 30 mg/kg) every other day to male and female mice prior to and during mating and continuing in females throughout organogenesis resulted in no adverse effects on pre- or postnatal development.
Plasma exposures at the highest dose tested (30 mg/kg) were approximately 4 times that in humans at the RHD. 8.2. Lactation Risk Summary There are no data on the presence of tofersen or its metabolites in human milk, the effects on the breastfed infant, or the effects on milk production.
Tofersen was detected in the milk of lactating mice following subcutaneous administration. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for QALSODY and any potential adverse effects on the breastfed infant from QALSODY or from the underlying maternal condition. 8.4.
Pediatric Use Safety and effectiveness in pediatric patients have not been established. 8.5. Geriatric Use A total of 13.5% (22/162) patients were 65 years of age and older and 1.2% (2/162) patients were 75 years of age and older at initiation of treatment in clinical studies for ALS in patients who have a mutation in the superoxide dismutase 1 ( SOD1 ) gene [see Clinical Studies ( 14 )] .
No overall differences in safety or effectiveness were observed between these patients and younger patients, but a greater sensitivity of some older individuals cannot be ruled out. There is no evidence for special dosage considerations based on age when QALSODY is administered.
🤰 Pregnancy ▾
8.1. Pregnancy Risk Summary There are no adequate data on developmental risks associated with the use of QALSODY in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively.
The background risk of major birth defects and miscarriage for the indicated population is unknown. Data Animal Data Subcutaneous administration of tofersen (0, 3, 10, 30 mg/kg) every other day to pregnant mice during the period of organogenesis resulted in no adverse effects on embryofetal development. Plasma exposure at the highest dose tested (30 mg/kg) was approximately 4 times that in humans at the recommended human dose (RHD) of 100 mg.
Subcutaneous administration of tofersen (0, 3, 10, 30 mg/kg) every other day to pregnant rabbits during the period of organogenesis resulted in no adverse effects on embryofetal development. Plasma exposure at the highest dose tested (30 mg/kg) was approximately 20 times that in humans at the RHD. Subcutaneous administration of tofersen (0, 3, 10, or 30 mg/kg) every other day to male and female mice prior to and during mating and continuing in females throughout organogenesis resulted in no adverse effects on pre- or postnatal development.
Plasma exposures at the highest dose tested (30 mg/kg) were approximately 4 times that in humans at the RHD.
🧒 Pediatric Use ▾
8.4. Pediatric Use Safety and effectiveness in pediatric patients have not been established.
🧓 Geriatric Use ▾
8.5. Geriatric Use A total of 13.5% (22/162) patients were 65 years of age and older and 1.2% (2/162) patients were 75 years of age and older at initiation of treatment in clinical studies for ALS in patients who have a mutation in the superoxide dismutase 1 ( SOD1 ) gene [see Clinical Studies ( 14 )] . No overall differences in safety or effectiveness were observed between these patients and younger patients, but a greater sensitivity of some older individuals cannot be ruled out.
There is no evidence for special dosage considerations based on age when QALSODY is administered.
🧬 Clinical Pharmacology ▾
12 CLINICAL PHARMACOLOGY 12.1. Mechanism of Action Tofersen is an antisense oligonucleotide that causes degradation of SOD1 mRNA through binding to SOD1 mRNA, which results in a reduction of SOD1 protein synthesis. 12.2.
Pharmacodynamics Effect of Tofersen on Total CSF SOD1 Protein Total CSF SOD1, an indirect measure of target engagement, was evaluated in Study 1 Part C in SOD1-ALS patients [see Clinical Studies ( 14 )] . At Week 28 in Study 1 Part C, a reduction in total CSF SOD1 protein of 35% (geometric mean ratio to baseline) in the tofersen-treated group versus a 2% decrease from baseline in the corresponding placebo subjects in the ITT population was observed (difference in geometric mean ratios for tofersen to placebo: 34%; nominal p<0.0001).
Effect of Tofersen on Neurofilament Proteins Plasma NfL, a blood-based biomarker of axonal injury and neurodegeneration, was evaluated in Study 1 Part C in SOD1-ALS patients [see Clinical Studies ( 14 )] . At Week 28 in Study 1 Part C, mean plasma NfL was reduced 55% (geometric mean ratio to baseline) in the QALSODY-treated subjects, compared to a 12% increase with placebo in ITT population (difference in geometric mean ratios for QALSODY to placebo: 60%; nominal p<0.0001). Plasma NfL declined until approximately Day 113, after which the reductions were sustained.
The reductions in phosphorylated neurofilament heavy chain (pNfH) were similar compared to reductions in NfL, as were reductions in CSF compared to plasma. Cardiac Electrophysiology At the maximum approved recommended dosing regimen, QALSODY does not prolong the QTc interval to any clinically relevant extent. 12.3.
Pharmacokinetics Absorption Intrathecal administration of QALSODY into the CSF allows tofersen to be distributed from the CSF to central nervous system tissues. The maximum CSF trough concentration occurred at the third dose, which was the last dose of the loading period. There was little to no accumulation for CSF tofersen with monthly dosing after the loading phase.
Tofersen is transferred from CSF into the systemic circulation, with median time to maximum concentration (T max ) plasma values ranging from 2 to 6 hours. There was no accumulation in plasma tofersen exposure following monthly maintenance dosing. Distribution Autopsy tissue from patients treated with tofersen (n=3) showed that tofersen administered intrathecally was distributed within the central nervous system tissues.
Elimination Metabolism Tofersen is expected to be metabolized through exonuclease (3'- and 5')-mediated hydrolysis and is not a substrate for, or inhibitor or inducer of CYP450 enzymes. Excretion The primary route of elimination has not been characterized. The effective half-life in CSF is estimated to be 4 weeks.
Drug Interaction Studies No clinical drug interaction studies have been performed. In vitro, QALSODY is not a substrate or inhibitor/inducer of major CYP enzymes or a substrate or inhibitor of major transporters. Specific Populations Effect of sex, race, age, and body weight on tofersen exposure in plasma was not clinically significant.
The effect of these factors on tofersen exposure in CSF is unknown. Patients with Renal or Hepatic Impairment No clinical studies have been conducted to evaluate the pharmacokinetics of tofersen in patients with renal or hepatic impairment. Tofersen is not expected to undergo metabolism by hepatic enzymes.
12.6. Immunogenicity As with all therapeutic oligonucleotides, there is a potential for immunogenicity. Immunogenicity assay results are highly dependent on the sensitivity and specificity of the assay and may be influenced by several factors such as: assay methodology, sample handling, timing of sample collection, concomitant medications, and underlying disease.
For these reasons, comparison of the incidence of antibodies to tofersen with the incidence of antibodies to other products may be misleading. The immunogenic response to QALSODY was evaluated in 166 patients with post-baseline…
🧬 Mechanism of Action ▾
12.1. Mechanism of Action Tofersen is an antisense oligonucleotide that causes degradation of SOD1 mRNA through binding to SOD1 mRNA, which results in a reduction of SOD1 protein synthesis.
📦 How Supplied / Storage and Handling ▾
16 HOW SUPPLIED/STORAGE AND HANDLING 16.1. How Supplied QALSODY injection is a sterile, clear and colorless to slightly yellow solution supplied as 100 mg/15 mL (6.7 mg/mL) solution in a single-dose glass vial free of preservatives. The NDC is 64406-109-01.
16.2. Storage and Handling Store refrigerated between 2°C to 8°C (36°F to 46°F) in the original carton to protect from light. Do not freeze.
If no refrigeration is available, QALSODY may be stored in its original carton, protected from light at or below 30°C (86°F) for up to 14 days. If removed from the original carton, unopened vials of QALSODY can be removed from and returned to the refrigerator, if necessary, for not more than 6 hours per day at or below 30°C (86°F) for a maximum of 6 days (36 hours).
16.1. How Supplied QALSODY injection is a sterile, clear and colorless to slightly yellow solution supplied as 100 mg/15 mL (6.7 mg/mL) solution in a single-dose glass vial free of preservatives. The NDC is 64406-109-01.
📦 Storage and Handling ▾
16.2. Storage and Handling Store refrigerated between 2°C to 8°C (36°F to 46°F) in the original carton to protect from light. Do not freeze.
If no refrigeration is available, QALSODY may be stored in its original carton, protected from light at or below 30°C (86°F) for up to 14 days. If removed from the original carton, unopened vials of QALSODY can be removed from and returned to the refrigerator, if necessary, for not more than 6 hours per day at or below 30°C (86°F) for a maximum of 6 days (36 hours).
📋 Description ▾
11 DESCRIPTION Tofersen, an antisense oligonucleotide, is a 20-base residue (20-mer) 5-10-5 MOE gapmer mixed backbone oligonucleotide. Of the nineteen internucleotide linkages, fifteen are 3′-O to 5′-O phosphorothioate diesters, and four are 3′-O to 5′-O phosphate diesters. Ten of the twenty sugar residues are 2-deoxy-D-ribose and the remainder are 2′-O-(2-methoxyethyl)-D-ribose (MOE).
The residues are arranged so that there are five MOE nucleosides at the 5′ and 3′-ends of the molecule flanking a gap of ten 2′-deoxynucleosides. The cytosine and uridine bases are methylated at the 5-position. The structural formula is: Figure 1: Structural Formula for Tofersen The molecular formula is C 230 H 317 N 72 O 123 P 19 S 15 and the molecular weight is 7127.86 atomic mass units (amu).
QALSODY is supplied as a sterile, preservative-free, clear, and colorless to slightly yellow solution in a Type I glass vial to be administered by intrathecal administration. Each vial of drug product contains a single dose of 100 mg tofersen at a concentration of 6.7 mg/mL in a formulation containing 0.21 mg/mL calcium chloride dihydrate, 0.11 mg/mL dibasic sodium phosphate, 0.16 mg/mL magnesium chloride hexahydrate, 0.03 mg/mL monobasic sodium phosphate, 0.22 mg/mL potassium chloride, 8.77 mg/mL sodium chloride, and water for injection.
The pH of QALSODY is approximately 7.2 (range 6.7 to 7.7). Figure 1
💬 Information for Patients ▾
17 PATIENT COUNSELING INFORMATION Myelitis and/or Radiculitis Inform patients and caregivers that QALSODY could cause myelitis and radiculitis. Instruct patients and caregivers to contact their healthcare provider if symptoms consistent with these adverse reactions develop [see Warnings and Precautions ( 5.1 )] . Papilledema and Elevated Intracranial Pressure Inform patients and caregivers that QALSODY could cause papilledema and elevated intracranial pressure.
Instruct patients and caregivers to contact their healthcare provider if symptoms consistent with these adverse reactions develop [see Warnings and Precautions ( 5.2 )] . Aseptic Meningitis Inform patients and caregivers that QALSODY could cause aseptic meningitis. Instruct patients and caregivers to contact their healthcare provider if symptoms consistent with meningitis develop [see Warnings and Precautions ( 5.3 )].
57294-01 Manufactured by: Biogen MA Inc. Cambridge, MA 02142 QALSODY is a trademark of Biogen MA Inc. © Biogen Inc.