NeurologyLive® Year in Review 2022: Most-Read FDA Approval Stories

These were the most-read FDA approval stories that were part of our coverage in 2022, brought to you as part of NeurologyLive®'s Year in Review.

NeurologyLive® Year in Review 2022

Each year, the NeurologyLive® team tracks the progress of hundreds of therapeutic potentials through the clinical development pipeline. Eager to update the clinical community on the available options for the treatment of neurologic diseases, the staff is quick to provide the latest on FDA decisions that add to that collection of therapies. Aligned with the various conversations with experts on the clinical trial data on these therapies, the team provides coverage the moment these decisions happen.

In 2022, particularly, there was much FDA activity to track—there were many agency decisions, advisory committee meetings, and application submissions and acceptances, among other notable undertakings. Some patient populations saw first-ever approvals, while others were granted access to more convenient administration modalities or new formulations. Rare diseases reported therapeutic advances that had been years in the making, and a number of devices and digital platforms that provide new tools to clinicians received clearance from the agency—and all the while, our team was there to share the news.

Here, we'll highlight the most-viewed FDA coverage that appeared on NeurologyLive® this year. Click the buttons to read more about these agency decisions.

1. AMX0035 Approved for the Treatment of ALS

SEPTEMBER 29, 2022 — The FDA approved Amylyx Pharmaceuticals’ AMX0035, a coformulation of sodium phenylbutyrate-taurursodiol, for the treatment of amyotrophic lateral sclerosis (ALS). The drug, which will be marketed as Relyvrio, becomes the third approved therapy to help slow disease progression or mortality in ALS, following riluzole (Rilutek) in 1995 and edaravone (Radicava; MT Pharma) in 2017.

"Today’s decision by the FDA to approve RELYVRIO (sodium phenylbutyrate and taurursodiol; previously known as AMX0035), for the treatment of ALS in adults is an exciting milestone for Amylyx, representing our first regulatory approval in the US and our second regulatory approval worldwide, and importantly, the broader ALS community, including people living with ALS, their families, and clinicians."
– Josh Cohen and Justin Klee
Cofounders and co-COOs, Amylyx Pharmaceuticals
AMX0035 Approved for the Treatment of ALS

2. FDA Approves Vutrisiran to Treat hATTR Amyloidosis

JUNE 14, 2022 — After the FDA first extended the review period by an additional 3 months, the agency has approved Alnylam’s investigational subcutaneous RNA interface therapy vutrisiran, marketed as Amvuttra, for the treatment of transthyretin-mediated (ATTR) amyloidosis. The RNAi therapeutic administered once every 3 months was approved based on positive 9-month results from the phase 3 HELIOS-A study (NCT03759379). Results from the study showed that the treatment met the primary end point of change from baseline in the modified Neuropathy Impairment Score at 9 months.

"Today, Amvuttra has the potential to change the standard of care for people living with the polyneuropathy of this devastating disease. As the fifth RNAi therapeutic developed by Alnylam to receive regulatory approval in less than four years, we believe Amvuttra represents an important milestone that brings us one step closer to achieving our P5x25 goals aimed at Alnylam’s transition to a leading biotech company."
– Yvonne Greenstreet, MBChB
CEO, Alnylam Pharmaceuticals
FDA Approves Vutrisiran to Treat hATTR Amyloidosis

3. Ganaxolone Wins Landmark Approval for CDKL5 Deficiency Disorder

MARCH 21, 2022 — The FDA has approved ganaxolone (Ztalmy) for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder, also known as CDD, in patients aged 2 years and older. The oral suspension therapy is the first approved for CDD, according to Marinus Pharmaceuticals. Following its scheduling by the Drug Enforcement Administration, the therapy became available via a specialty pharmacy in July 2022.

“There has been a great unmet medical need for treatments that address seizures associated with CDKL5 deficiency disorder given their prominent role and profound impact on patients. To date, antiseizure treatment decisions have been based on very limited clinical evidence in this patient population and the resulting outcomes underscore the need for therapies that further improve seizure control.”
– Scott Demarest, MD
Neurologist and clinical director of precision medicine, Children’s Hospital Colorado
Ganaxolone Now Available After Winning Landmark Approval for CDKL5 Deficiency Disorder

4. FDA Approves Daridorexant for Insomnia Treatment

JANUARY 10, 2022 — The FDA has approved Idorsia’s daridorexant, a dual orexin receptor antagonist, for the treatment of insomnia in adults. Marketed under the name Quviviq, the medication, which has been recommended as a controlled substance by the FDA, is expected to be available in May 2022 following scheduling by the US Drug Enforcement Administration. Data showed that treatment with daridorexant 25- and 50-mg resulted in significant improvement compared with placebo on objective measures of sleep onset and sleep maintenance, as well as patient-reported total sleep time.

"After more than 20 years of research and a progressive understanding of the role of orexin in sleep-wake balance and of the potential of orexin receptor antagonism, we designed daridorexant to help address several issues people with insomnia face. Daridorexant properties include a potent inhibition of both orexin receptors, a rapid absorption for sleep onset, and a pharmacokinetic profile such that around 80% of daridorexant has been eliminated after a night of sleep to help minimize residual effects."
– Martine Clozel, MD
Chief scientific officer, Idorsia
FDA Approves Daridorexant for Insomnia Treatment

5. FT218 Receives Tentative Approval for EDS, Cataplexy in Adults With Narcolepsy

JULY 20, 2022 — The FDA has granted tentative approval to Avadel Pharmaceuticals’ extended-release oral suspension formulation of sodium oxybate, marketed as Lumryz, for the treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy. Previously, the therapy was known as FT218. The new drug application was supported by data from the phase 3 REST-ON study (NCT02720744), which was held under a special protocol assessment agreement with the FDA.

“We have reached a critical milestone, as tentative approval confirms the safety profile and clinical efficacy of LUMRYZ for adults with narcolepsy. Tentative approval is an important regulatory step forward and indicates LUMRYZ could potentially be granted final approval in 11 months or less. We believe once-at-bedtime LUMRYZ offers the opportunity to positively transform the lives of oxybate-eligible patients living with narcolepsy. Our extensive market research indicates Avadel is well-positioned to capture a significant share of the oxybate-eligible patient population which we estimate to be in excess of 30,000 patients. We are pursuing all options to accelerate final approval on or before June 2023 and prepare for commercial launch."
– Greg Divis
CEO, Avadel Pharmaceuticals
FT218 Receives Tentative Approval for EDS, Cataplexy in Adults With Narcolepsy

6. FDA Approves Risdiplam to Treat Presymptomatic Babies Under 2 Months With SMA

MAY 31, 2022 — The FDA has approved an expanded indication for risdiplam (Evrysdi; Genentech) to include the treatment of presymptomatic babies under 2 months old with spinal muscular atrophy (SMA), making it the first approved treatment administered at home for this patient group. Risdiplam is now approved to treat SMA in both children and adults of all ages.

"The approval of Evrysdi for presymptomatic babies is particularly important, as early treatment of SMA, before symptoms start to arise, can help babies to achieve motor milestones. With the inclusion of SMA in newborn screening programs, this approval provides the opportunity to start treating at home with Evrysdi soon after the diagnosis is confirmed."
– Richard Finkel, MD
Director of the experimental neuroscience program, St. Jude Children's Research Hospital
FDA Approves Risdiplam to Treat Presymptomatic Babies Under 2 Months With SMA

7. Roche Gets FDA Clearance for CSF Beta-Amyloid and Phospho-Tau Alzheimer Disease Assays

DECEMBER 9, 2022 — According to an announcement from Roche, the company’s Alzheimer disease cerebrospinal fluid Elecsys assays—including beta-amyloid1-42 CSF II (Abeta42) and phospho-Tau181P (pTau181)—have been approved by the FDA for use with the cobas fully automated immunoassay analyzers. The biomarkers are hallmarks of AD pathology, and the assays utilize their ratio (pTau181/Abeta42) for measurements. Roche noted in the announcement that its AD assays “achieve 90% concordance” with amyloid PET scans.

“Globally, up to 75% of people living with Alzheimer’s disease have not been diagnosed, and those who have often report a long and complicated process. The Elecsys AD CSF assays have the potential to guide more people with suspected Alzheimer’s disease toward a diagnosis than ever before. As we are starting to see exciting results for new potential Alzheimer’s treatments, reliable tests that have been clinically validated will be critical in ensuring the right patients are identified and able to benefit from them.”
– Thomas Schinecker, PhD
CEO, Roche Diagnostics
Roche Gets FDA Clearance for CSF Beta-Amyloid and Phospho-Tau Alzheimer Disease Assays

8. FDA Approves Midazolam Autoinjector for Status Epilepticus

AUGUST 30, 2022 — The FDA has given the green light to Rafa Laboratories for its 10-mg midazolam autoinjector for the treatment of status epilepticus in adults. It is the first autoinjectable product to be approved by the FDA, joining other rescue treatments with different routes of administration. The midazolam autoinjector is administered into the thigh, and can be done through clothing, without requiring an intravenous line, according to Rafa.

"The usability of the autoinjector has a significant medical advantage in its immediate treatment effect, as well as in reducing long-term damage. Clinical studies confirmed the correlation between early treatment of status epilepticus and a reduced risk of an ongoing and irreversible neurological damage. This product could assist in saving many lives around the globe."
– Roy Shay
Head of emergency solutions, Rafa Laboratories
FDA Approves Midazolam Autoinjector for Status Epilepticus

9. Parkinson Disease Symptom Data Tool StrivePD Receives FDA Clearance

JUNE 21, 2022 — The FDA has granted 510(k) clearance to Rune Labs’ StrivePD software ecosystem, an application that collects data on symptoms of patients with Parkinson disease (PD) using an Apple Watch, with the intention of improving care management and clinical trial design. The hope is that StrivePD may help locate and facilitate patients with prodromal PD, the stage at which individuals do not fulfill the diagnostic criteria for PD but do exhibit signs and symptoms.

"As we have seen in oncology, the introduction of large quantities of real-world data has the power to transform drug development and fundamentally change disease prognosis. This clearance is a major step towards building a similar paradigm in neurology. With all of the data we will collect and the patients we will reach through this clearance, we will make sure the right participants enroll in trials, and help our pharma and medtech partners run more efficient trials with higher quality outcomes data, thereby enabling more therapies to come to market quickly to help those suffering from Parkinson's."
– Brian Pepin, MSEE
CEO and founder, Rune Labs
Parkinson Disease Symptom Data Tool StrivePD Receives FDA Clearance

10. Zonisamide Oral Suspension FDA-Approved for Partial Seizures in Epilepsy

JULY 18, 2022 — Originally approved in the US in 2000, the FDA has cleared a new oral suspension of zonisamide (Azurity Pharmaceuticals), marketed as Zonisade, for the treatment of partial seizures in adults and pediatrics aged 16 years and older with epilepsy. Designed as a carbonic anhydrase inhibitor, the efficacy and tolerability of the oral suspension had been previously established in 3 double-blind, placebo-controlled, multicenter trials. Additionally, the drug is contraindicated in patients who have demonstrated hypersensitivity to sulfonamides or zonisamide.

"The provider community continues to seek reliable formulations of medicines that may reduce the epilepsy patient and caregiver burden and help improve treatment adherence. Zonisade addresses an important unmet need in patients who have difficulty swallowing or who are unable or unwilling to take tablets."
– James Wheless, MD
Chair of pediatric neurology, University of Tennessee Health Science Center
Zonisamide Oral Suspension FDA-Approved for Partial Seizures in Epilepsy

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