Govorestat linked to functional Improvement in sorbitol dehydrogenase deficiency, findsTrial

Govorestat was found to be clinically beneficial for patients with sorbitol dehydrogenase (SORD) deficiency, according to interim results from the phase 3 INSPIRE trial.

The INSPIRE trial is a Phase 3 double-blind placebo-controlled registrational study evaluating the effect of once-daily (QD) oral govorestat (AT-007) in 56 patients aged 16-55 with SORD Deficiency in the US and Europe.

SORD Deficiency is a debilitating, hereditary axonal neuropathy caused by mutations in the Sorbitol Dehydrogenase gene, leading to an inability to metabolize the sugar sorbitol and resulting in accumulation of high levels of toxic sorbitol, which causes motor neuron degeneration and loss of mobility and motility. Govorestat is a central nervous system penetrant Aldose Reductase Inhibitor, which blocks the conversion of glucose to sorbitol, and has previously been shown to reduce sorbitol levels in patients with SORD Deficiency.

The objective of this pre-specified, 12-month interim analysis was to evaluate early indicators of govorestat treatment effect in order to inform future regulatory discussions and support a potential New Drug Application (NDA) submission, due to the urgent need for treatment and absence of any other options for patients with SORD Deficiency. The 12-month interim analysis was comprised of a clinical efficacy primary endpoint based on correlation of sorbitol with composite clinical outcome measures, and a pharmacodynamic (PD) biomarker primary endpoint based on sorbitol reduction.

Interim Analysis Results:

  • Demonstrated statistically significant correlation between sorbitol level and the prespecified CMT-FOM composite clinical endpoint (10-meter walk-run test, 4 stair climb, sit to stand test,6-minute walk test and dorsiflexion) (p=0.05).
  • Govorestat treatment provided sustained reduction in sorbitol level in patients with SORD Deficiency over 12 months of treatment, which was statistically significant compared to placebo (p<0.001).
  • Govorestat treatment also resulted in a highly statistically significant effect (p=0.01) on the CMT Health Index (CMT-HI), an important patient-reported outcome measure of disease severity and well-being, which was a secondary endpoint in the study. Aspects of the CMT-HI that demonstrated a treatment effect included lower limb function, mobility, fatigue, pain, sensory function, and upper limb function.
  • Govorestat was safe and well tolerated, with similar incidence of adverse events between active and placebo-treated groups.

We believe the results from the 12-month interim analysis confirm the role of sorbitol as a key driver of disease severity and progression over time. Clinical outcomes of the ongoing INSPIRE trial are expected to be assessed again at 24 months, where the 10-meter walk run test serves as the primary clinical efficacy endpoint. The Company plans to discuss a potential NDA submission with the U.S. Food and Drug Administration (FDA) based on the clinical data to date.

“Our commitment to bringing first of their kind therapies to rare disease indications with no existing treatment options is at the core of our work,” said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics. “We are excited by the prospect of providing patients with SORD Deficiency with a treatment option that has the potential to slow disease progression and the consistent benefit demonstrated by govorestat.”

“We are thrilled by the results of this 12-month interim analysis, which demonstrate govorestat’s effectiveness in reducing sorbitol levels and improving key functional measures and patient reported outcomes in SORD Deficiency, including lower limb function, upper limb function, fatigue and pain,” said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. “We look forward to meeting with regulatory agencies regarding a path to potential approval based on this data, and endeavor to bring this important treatment to patients as quickly as possible.”

“As a neurologist and neuromuscular specialist, I am delighted to see such strong results from just 12 months of treatment with govorestat for this debilitating disease with no existing treatment options available,” said Michael Shy, MD, Director of the Division of Neuromuscular Medicine at Carver College of Medicine, University of Iowa Medical Center, and Principal Investigator on the INSPIRE Phase 3 trial. “The results from this interim analysis have exceeded my expectations, with a statistically significant impact on how patients feel and function, as measured by the CMT-HI patient reported outcome measure. The ability to reduce sorbitol levels, which we believe to be the pathogenic cause of damage in these patients, coupled with standardized metrics of patient function and well-being are strong indicators of treatment benefit.”

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Health Ministry collaborates with QCI to Improve Healthcare Quality in CGHS Hospitals

New Delhi: Aiming to transform the Central Government Health Scheme (CGHS) ecosystem and improve the healthcare experience for all beneficiaries, the Union Ministry of Health and Family Welfare announced a collaboration with the Quality Council of India.

A press release issued in this regard stated that this partnership aims to deliver a more robust, efficient, and transparent CGHS system by outlining a comprehensive approach that will address several key areas.

The collaboration will be focussed on enhancing the quality of services offered through systematic interventions. The MoU aims to support CGHS facilities in achieving accreditation by NABH/NABL and to build the capacity of healthcare providers to deliver high-quality services, reports ANI.

The agreement emphasizes the importance of technology in achieving the desired transformation and lays the groundwork for collaboration on additional initiatives to further improve the CGHS ecosystem.

Measures to ensure the quality of drugs and storage facilities, alongside modernization of procurement and inventory management systems will also be a crucial aspect along with a focus on the modernization of infrastructure, products, and processes.

Furthermore, to ensure regular review and evaluation, quality monitoring mechanisms will be established. With an aim to provide a complete overhaul of the CGHS system through third-party assessments and the implementation of transformation initiatives, this collaboration will also focus on expanding the network of qualified healthcare providers and laboratories empanelled under CGHS.

Also Read: Either Govt fixes standard rates for private hospitals or we will implement CGHS rates: Supreme Court issues Ultimatum

Manashvi Kumar, the Joint Secretary of the Union Health Ministry, said, “This is a historic moment. The collaboration marks the beginning of a quality overhaul of the CGHS ecosystem which has commitment from the highest level of the Government of India. CGHS must become the role model for healthcare in India.”

Jaxay Shah, the Chairperson of the Quality Council of India, highlighted this collaboration as a pivotal moment that will usher in a new era of quality, transparency, and efficiency for the CGHS ecosystem.

“By leveraging QCI’s expertise in quality improvement methodologies and the Ministry’s deep understanding of the healthcare landscape, we can implement a comprehensive transformation plan. This will not only enhance service delivery but also empower beneficiaries with greater trust and confidence in the CGHS system,” he stated.

Rajesh Maheshwari, the Secretary General of QCI and CEO of National Accreditation Board for Certification Bodies said that this five-year partnership represents a significant step forward in our collective vision of ensuring high-quality, accessible healthcare for all Indians.

“By focusing on quality accreditation, capacity building, and technology integration, we are laying the groundwork for a sustainable system that can adapt and evolve to meet the ever-changing needs of beneficiaries. This collaboration will empower us to leverage innovation and best practices to create a CGHS that is not only efficient but also empowers patients to take an active role in their healthcare journey,” Maheshwari said.

This move comes at a time when recently the Supreme Court cognisance of the disparity of the cost of services at government and private hospitals while considering a Public Interest Litigation (PIL) filed by an NGO ‘Veterans Forum for Transparency in Public life’. Filing the plea under Article 32 of the Constitution, the NGO prayed for a direction on the Union Government to determine the rate of fee chargeable from the patients in terms of Rule 9 of the Clinical Establishment (Central Government) Rules, 2012.

Even though Rule 9 of the Clinical Establishment (Central Government) Rules, 2012 mandates that the Government shall determine the rate of fee chargeable from the patients at private hospitals and clinical establishments, it has not been enforced till now.

Rule 9 states, “every clinical Establishment shall display the rates charged for each type of service provided and facilities available, for the benefit of the patients at a conspicuous place in the local as well as english language.” It further mentions that the clinical establishments shall charge the rates for each type of procedures and services within the range of rates determined and issued by the Central government from time to time, in consultation with the State governments.

Slamming the Union Government for its failure to fix a range of rates of services at private healthcare facilities. Issuing directions to the Union Health Secretary to hold a meeting with the State Counterparts and come up with a concrete proposal before the next date of hearing, the Supreme Court bench had warned that if the Government fails to comply with the directions, the Court shall consider issuing CGHS rates instead.

Also Read: Standardising rates for healthcare: Health Ministry Calls Emergency Meeting with Private Hospitals

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Charges increased from Rs 35k to Rs 75k: MGIMS medicos file complaint over Sudden Mid-Course Fee Hike

Mumbai: The medical students enrolled at Wardha-based Mahatma Gandhi Institute of Medical Sciences (MGIMS) are upset over the sudden mid-course hike in the college fees and hostel charges. While for the academic year 2022-2023, the students had to pay Rs 35,000 as additional charges, they are now required to pay Rs 75,800 for the academic year 2023-2024. A complaint in this regard has been filed with the state medical education department.

Students complained that in the middle of their four-and-a-half-year course, the college levied additional charges and also increased the hostel fees without providing any additional facilities. Further, the students alleged that the medical college is insisting on compulsory enrolment in the hostel, even for the interns, in contradiction of the directives issued by the National Medical Commission (NMC). As per the rules prescribed by the Apex Medical Commission, even though all institutes have to offer accommodation, it cannot be made mandatory for the students.

Alleging that the college administration has ‘unlawfully increased their college fee’, a complaint was submitted earlier this month to the Medical Education Department of the State Government, reports TOI.

MGIMS is managed by the Central Government, the State, and the Kasturba Health Society and these authorities share its expenditure in the proportion of 50%, 25%, and 25%, respectively. Since the college charges a nominal fee, it is one of the sought-after institutes for the medical aspirants. The students are required to pay a tuition fee of Rs 1 lakh, almost the same as the government medical colleges.

Also Read: Fee Hike but not Stipend Hike: Kerala PG doctors to go on 24 hour strike on 8th November

As per the latest media report by the Times of India, some of the students are now complaining that the college has levied additional charges in the middle of their MBBS course and also hiked their hostel fees without offering any extra facilities. The hostel fee was increased in July last year from Rs 12,500 to Rs 30,000 per semester. Now, the fee has been hiked by 65% for the interns. While previously, the interns had to pay Rs 3000 per month, they are now required to pay Rs 5000 from April. 

The Complaint, submitted to the State Medical Education Department, mentioned that the college started charging Rs 4,500 for Wi-Fi and Rs 4,800 for transportation per semester.

In this regard, a student alleged that the additional charges have been levied now even though the college had been offering Wi-Fi facilities since day one. The student further mentioned that library charges, electricity charges, and even mess charges have been almost doubled. As a result, the total additional charges, which were Rs 35,000 per semester in 2022-2023, has now become Rs 75,800 in 2023-2024.

“Recently, the stipends for medical interns were hiked in all colleges from Rs 11,000 to Rs 18,000, but the college has simultaneously increased additional fees, so effectively students are still getting the old stipend amount,” the student mentioned.

Addressing the issue, Counsellor Jasmine Gogri pointed out that as per the government rules, fees charged in the first-year should remain unchanged for the entire course. “Most colleges, however, are charging additional fees under different heads, but this is a government college,” Gogri added.

Meanwhile, commenting on the matter, the secretary of State Medical Education Department Dinesh Waghmare informed that they would look into the complaint.

Also Read: Fee for MBBS hike by Rs 1 lakh in Maharashtra private medical colleges this year

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Reduce PSA level to less than 4 in inclusion criteria: CDSCO Panel Tells Akum Drugs and Pharmaceuticals on FDC Dutasteride plus Silodosin

New Delhi: Reviewing the active post-marketing surveillance (PMS) protocol of fixed-dose combination (FDC) Dutasteride plus Silodosin presented by Akums Drugs and Pharmaceuticals, the Subject Expert Committee (SEC) functional under the Central Drug Standard Control Organisation (CDSCO) has opined the firm to modify inclusion criteria by reducing the Prostate-Specific Antigen (PSA) level to less than 4 and to include more geographically distributed sites.

In addition to the above, the expert panel suggested that PSA should be done at baseline and at the end of the study after 3 months. Furthermore, the committee advised to include more geographically distributed sites so that there should be 50% each from government and private sites.

This came after Akums Drugs and Pharmaceuticals presented the active post-marketing surveillance (PMS) protocol before the committee, in light of the condition mentioned in permission in Form CT-23 dated 09.12.2022.

Dutasteride is in a class of medications called 5-alpha reductase inhibitors. It works by blocking the production of a natural substance that enlarges the prostate. Dutasteride blocks the action of an enzyme called 5-alpha-reductase. This enzyme changes testosterone to another hormone that causes the prostate gland to grow. Dutasteride will cause the size of the prostate to decrease, but the effect lasts only as long as the medicine is taken.

Silodosin is in a class of medications called alpha-blockers. It relieves the symptoms of BPH by relaxing the muscles of the bladder and prostate. Silodosin is used to treat signs and symptoms of an enlarged prostate gland, which is also known as benign enlargement of the prostate (benign prostatic hyperplasia or BPH). Benign enlargement of the prostate is a problem that can occur in men as they get older. The prostate gland is located below the bladder.

At the recent SEC meeting for Urology held on 27th March 2024, the expert panel reviewed the active PMS protocol of the Dutasteride plus Silodosin (0.5mg/0.5mg + 4mg/8mg) uncoated bilayered tablet.

After detailed deliberation, the committee opined the following modification:

1. Inclusion criteria should be modified by reducing the PSA level to less than 4.

2. PSA should be done at baseline and at the end of the study after 3 months.

3. More sites to be included which should be geographically distributed so that there should be 50% each from Government and Private sites.

Accordingly, the expert panel stated that a revised Active PMS protocol should be submitted to CDSCO for further review by the committee.

Also Read: CDSCO Panel Approves J&J’s Protocol Amendment Proposal For Amivantamab, Lazertinib combination

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USFDA successfully concludes inspection at Piramal Pharma USA facility

Mumbai: Piramal Pharma Limited has informed through a recent BSE filing that the US Food and Drug ADministration (US FDA) has
issued an Establishment Inspection Report (EIR) for the Riverview, USA manufacturing facility and the
inspection has now been successfully closed by the US FDA.

Medical Dialogues team had earlier reported that the US FDA had concluded a Pre-Approval Inspection (PAI) with 3 observations at the Company’s Riverview (USA) facility.

Read also: Piramal Pharma gets 3 USFDA observations for US facility

Piramal Pharma Limited (PPL) offers a portfolio of differentiated products and services through end-to-end manufacturing capabilities across 15 global facilities and a global distribution network in over 100 countries. PPL includes Piramal Pharma Solutions (PPS), an integrated Contract Development and Manufacturing Organization; Piramal Critical Care (PCC), a Complex Hospital Generics business, and the India Consumer Healthcare business selling over-the-counter products.

PPS offers end-to-end development and manufacturing solutions through a globally integrated network of facilities across the drug life cycle to innovators and generic companies. PCC’s complex hospital product portfolio includes inhalation anaesthetics, intrathecal therapies for spasticity and pain management, injectable pain and anaesthetics, injectable anti-infectives, and other therapies.

In addition, PPL has a joint venture with Allergan. In October 2020, the company received a growth equity investment from the Carlyle Group.

Read also: Piramal Critical Care unveils new 10mg/10mL (1 mg/mL) concentration of Zinc Sulfate for Injection in US

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Laparoscopic Bilateral Adrenalectomy performed on 9-year-old girl at MKCG Medical College Hospital

Doctors at the government-run MKCG Medical College and Hospital recently achieved a milestone by performing a laparoscopic bilateral adrenalectomy on a 9-year-old girl suffering from Cushing syndrome, an exceptionally rare condition. This marked the first instance of such a procedure in the state.

Cushing syndrome is caused due to a rare genetic disease called primary pigmented nodular adrenocortical disease (PPNAD), an extremely rare disorder with a prevalence of less than one in two lakh. The doctors took around three hours to perform the laparoscopic on April 9, said a doctor.

For more details, check out the full story on the link below:

MKCG Medical College Hospital Doctors Perform Laparoscopic Bilateral Adrenalectomy On 9-Year-Old Girl

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First in Odisha: AIIMS Bhubaneswar launches Advanced Artificial Urinary Sphincter Implantation Service

Bhubaneshwar: Adding another feather to its cap trusted tertiary care national health institution, AIIMS Bhubaneswar is proud to announce the launch of a cutting-edge service for the placement of Artificial Urinary Sphincters (AUS), a significant advancement in urological care. This innovative service is set to provide new hope and an improved quality of life for patients suffering from severe urinary incontinence. This modern facility is first of its kind in Odisha. Earlier this implantation was only available in the metro cities of our country. 

The Artificial Urinary Sphincter (AUS) implantation procedure is recognized as a gold standard treatment for urinary incontinence that has not responded to conventional treatment modalities. This service is particularly tailored for patients who have experienced urinary incontinence due to prostate surgery, spinal cord injuries, or other underlying conditions that affect urinary control.  

Also Read:1.1 kg newborn with duodenal atresia gets new lease of life at AIIMS Bhubaneswar

Executive Director, Prof. (Dr.) Ashutosh Biswas congratulated the team of doctors and said, “The Department of Urology at AIIMS Bhubaneswar has consistently been providing world class care which is at the forefront of technology and innovation. With the introduction of the services of Artificial Urinary Sphincter implantation here, the people of Odisha and Eastern India will have access to this service, for which they had to travel to centres in other states earlier.”

The AUS program at AIIMS Bhubaneswar will be led by a team of highly skilled urologists and surgeons, trained in the latest techniques and using state-of-the-art technology. “The initiation of the AUS implantation service represents a pivotal step in our commitment to offering comprehensive and advanced medical treatments,” said Dr Sambit Tripathy, Assistant Professor, Department of Urology, involved in delivering Andrology care at AIIMS Bhubaneswar. Dr Prasant Nayak, Professor and Head of the Department of Urology added “We are dedicated to providing our patients with the best possible outcomes and improving their quality of life. We are thankful and indebted to the Director, Prof. (Dr.) Ashutosh Biswas, who has always encouraged us in our endeavour to bring the latest advances in urological care to the population of Odisha. We are also thankful to our patients for their immense trust and faith in us.”

Also Read:AIIMS Bhubaneswar extends support for Down Syndrome and other Genetic Disorders

The Medical Superintendent of AIIMS, Bhubaneswar congratulated and felicitated the doctors of the Department of Urology.

AIIMS Bhubaneswar has equipped itself with the latest medical technologies to ensure the highest standards of safety and efficacy for all procedures performed within this new service. The hospital also plans to conduct regular follow-up clinics and support groups to assist patients during their recovery process.

Patients seeking more information about the Artificial Urinary Sphincter implantation service at AIIMS Bhubaneswar or wishing to schedule an appointment can contact the Urology Department at AIIMS Bhubaneswar.  

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Smoking-related deaths could be reduced if people attending lung cancer screening are offered stop-smoking support

A new study has found that by offering stop-smoking support as part of the national lung cancer screening program, there is potential to save lives, and dedicated funding must be considered by policymakers.

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Study finds ChatGPT shows promise as medication management tool, could help improve geriatric health care

Polypharmacy, or the concurrent use of five or more medications, is common in older adults and increases the risk of adverse drug interactions. While deprescribing unnecessary drugs can combat this risk, the decision-making process can be complex and time-consuming. Increasingly, there is a need for effective polypharmacy management tools that can support short-staffed primary care practitioners.

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Opioid dependence remains high but stable in Scotland, new surveillance report finds

Opioid dependence in Scotland remains high but largely stable, according to a new University of Bristol-led analysis published in Addiction. The study is the first to estimate the number of people dependent on opioid drugs (such as heroin), and who are in or could benefit from drug treatment, among Scotland’s population since 2015/2016 estimates were published.

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